Advancing rare disease research with a patient-centered approach

What is the rare Gaucher disease and how does it impact patients, families, and life sciences? Is enough emphasis being placed on research and discovery for rare diseases? And what are the patient-centered approaches that best serve those battling rare diseases? We will get those answers and more in this episode with Tanya Collin-Histed, CEO of the International Gaucher Alliance. Tanya has been a longtime driving force in supporting patients with rare diseases and advocating for world-class healthcare. Her work has been nothing short of groundbreaking and she’s become the go-to person for patients, medical practitioners, industry, and governing bodies. As a mother of a child with Gaucher disease, she brings a unique, first-hand, and compassionate approach.   -------------------------------------------------------- Episode Transcript:  00;00;00;00 - 00;00;25;09  What is the rare gosh disease? Is enough emphasis being placed on rare diseases? And what are the patient centered approaches that best serve those battling rare diseases? We'll get those answers and more on research in action in the lead to the world. Hello and welcome to another episode of Research and Action, brought to you by Oracle Life Sciences.     00;00;25;09 - 00;00;49;25  I'm Mike Stiles. And today we have a truly inspiring guest. Tanya calling his dad, CEO of the International Gosh Alliance, has been a long time driving force in supporting patients with rare diseases and advocating for world class health care. Our work has been nothing short of groundbreaking. She's actually become quite the go to person for patients, medical practitioners, industry governing bodies.     00;00;50;03 - 00;00;52;01  Tanya, thanks so much for being with us today.     00;00;52;14 - 00;00;55;04  Thanks, Mike. It's an absolute pleasure to be here.     00;00;55;19 - 00;01;05;11  Well, before we get into the incredible work you're doing, let's get a baseline understanding of exactly what Gaucher disease is and just how rare it is.     00;01;06;00 - 00;01;37;11  Okay. Well, as a caregiver, I'll give a lay lay version to you. So it's a genetic condition and it's inherited. It's caused by a storage disorder. And that is because people with Gaucher have a deficiency in an enzyme. And the function of that enzyme is that it's in the body to break down substances. And because there isn't enough of that enzyme, the substances store in different parts of the body.     00;01;37;25 - 00;02;05;17  And it really does depend on what type of disease you have to how the disease affects you. But all patients can have a large liver and spleen. They get anemia, they get bruising where the blood doesn't clot properly and bone pain and bone damage due to the cells being in their bone marrow where which is where the blood cells are made.     00;02;06;02 - 00;02;42;09  Now, for patients who have type two and type three, there's also brain involvement and that really ranges from patient to patient. But that can include things like cognitive impairment, seizures, hearing and sight loss, unsteadiness in their movements and tremors. Now, it's it's a rare disease, as you say, and roughly it's around one in 100,000. However, this will different differ from region to region and also from type to type.     00;02;42;21 - 00;03;11;18  So historically, type one cases, disease is the most prevalent. Then we go into type three and then type two is like what we would call ultra ultra. However, as we become a much more globally connected community, we are seeing that there are many more patients with type two and Type three in Asia, whereas in sort of Europe and the West, we see more Type one patients.     00;03;12;05 - 00;03;27;29  Yeah, well it sounds like just that one issue, the the deficiency of that enzyme can cause countless problems all over the body. It already makes it obvious why this is such a difficult disease to get a handle on.     00;03;28;17 - 00;04;03;25  Yeah, absolutely. And I think the thing is, is that often when patients become ill and they go to maybe their general practitioner, you know, and they describe the, you know, how they feel that there are lots of things that could be wrong with patients. And therefore often patients have what we call a sort of diagnostic journey, a diagnostic odyssey where it will take a long period of time for them to actually get diagnosed.     00;04;04;12 - 00;04;12;04  If someone is diagnosed and they do get a correct diagnosis for, gosh, what are the typical outcomes?     00;04;12;20 - 00;04;38;28  Wow, that's a good question. So again, this goes back to whether or not you have type one, Type two or type three as a rare disease. We are incredibly lucky. So over 30 years ago, there was a medicine developed called enzyme replacement therapy, and this was developed and it what it does is it puts the deficient enzyme back into the patient's body.     00;04;39;06 - 00;05;00;14  So it's a bit like, you know, when you've been men. Tom So you've been, you know, you start up or your waist and, you know, you put it to one side and then the binmen come and they empty it, and then you start to store it up again. Well, of course, it's a bit man dotcom, you know, that storage gets more and more and more and starts to affect the average around it.     00;05;00;20 - 00;05;34;04  So that that's a sort of good analogy for go phase disease. But because this enzyme replacement therapy was developed and it was it's like an infusion. So patients either have it once a week or once a fortnight, it puts the enzyme back into the body, gets rid of all the storage and a significant proportion of patients. If they get treatment early on and they get the right dose of treatment, then they can actually live really good lives with great outcomes.     00;05;34;10 - 00;06;05;22  Now here it's important to say that enzyme replacement therapy is for the non neurological aspects of the disease. So that is your liver, your spleen, your bones. Now it doesn't cross the blood brain barrier. So the type for patients with type two and type three, they still have the all the neurological aspects of of the disease. So if you're type one, it will depend on where you live in the world, whether or not you get treatment.     00;06;05;22 - 00;06;13;28  And that's some issue. But if you do get treatment and you get good clinical care, then you can expect to have a relative normal life.     00;06;14;18 - 00;06;31;20  Well, and unfortunately, the reason the world has you as such a strong advocate is that this is a disease faced by your own daughter. Tell us about her, what her symptoms were when they started showing up and that journey that you mentioned of getting properly diagnosed and treated.     00;06;32;10 - 00;07;01;05  Of course, yeah. This is this is going back a few years ago now. So in 1995, Maddie was my daughter, Maddie was 15 months old. And it was towards the end of the year and we just noticed that she just wasn't that well. And she had quite a low mood and a cold. And, you know, like many pet parents, you know, she was was still quite young.     00;07;01;05 - 00;07;29;11  So we took her to the doctors and they were like, yeah, she's got a you know, she's got a throat infection, She's got ear infection. You know, hear the antibiotics go away If she doesn't get any better, come back. So a week goes by, ten days go by. She's she's not any better. So we took her back and at that point, the general practitioner said she's very pale, if you notice that she's very pale.     00;07;29;25 - 00;07;47;12  And we was like, Well, yeah, we have noticed, but that's why we just thought it was part of her not not feeling great. So he said, I'll tell you what he said, I think we should you should go to the local doctor, local hospital and they'll do a hemoglobin C what her, her blood types are, and we'll take it from there.     00;07;48;13 - 00;08;20;01  Well, that from that morning, basically, we went on a three month journey to the local hospital. Her hemoglobin was 6.4, where it should be around 12. She was admitted she had a number of blood transfusions. On examination, they found out she had a large liver and spleen. We were given the diagnosis of leukemia. So that was obviously very, very challenging for us as a family.     00;08;20;02 - 00;08;55;05  She was our first born. Now, at this point in time, we lived not far from London, and the local hospital had shared care for pediatric pediatric oncology with Great Ormond Street Hospital, who most people would have heard of. So we were taken by ambulance to Great Ormond Street Hospital. We were admitted onto the oncology ward and it was like a little conveyor belt of all these little children going through for Beaumaris to aspirations so that they could give her a final diagnosis.     00;08;56;05 - 00;09;23;16  Actually, after waiting a number of hours, we were told that she didn't have leukemia, but they suspected that she had something called Go Shay's Disease, which was a very rare disease. Now, you will remember I previously set about this diagnostic journey and diagnostic odyssey, and it takes a long time to be diagnosed. Now, ours was not a typical one for a patient with rare disease.     00;09;24;02 - 00;09;56;11  And it goes back to what I said about there being that new medicine in the early 1990s. And because it was approved, the company put investment into awareness and actually Great Ormond Street Hospital had become a center of excellence for Go Shay's Disease. And they had a very, very good doctor there. And actually that doctor cared for Maddie until she was 18 years old at Great Ormond Street when she transferred to the adult hospital at the Royal Free.     00;09;57;23 - 00;10;27;19  Now, you know, we were lucky because when they did that bone aspiration for leukemia, because of their expertise, they noticed the sort of shape and the pattern of the cell and that's why she was diagnosed with Go Shay's Disease. And actually from the first visit to the doctor at the end of 1995 to her first infusion of the new medicine to help with her liver and spleen, it was only actually approximately six weeks.     00;10;28;04 - 00;10;59;20  So we were, you know, we were very lucky. We did stay in Great Ormond Street for three months because her liver and spleen were so large. She underwent a what we call a partial splenectomy. So she had most of her spleen removed. She was severely underweight and her breathing was very shallow at the time of of admission. So we were in Great Ormond Street for a long time when she when she was first diagnosed.     00;10;59;20 - 00;11;15;06  And actually she was on a feeding tube for about a year afterwards just to build her back up. But, you know, if you can have a great diagnosis, then I think we were extremely lucky. In that case.     00;11;15;23 - 00;11;37;28  I would absolutely have to agree. I mean, what a horrible thing for Maddie to have to go through and for your family to have to go through. But, you know, it sounds like you landed in exactly the right place to be with the right people. And then based on my own experience, you know, it's not uncommon to go beyond just caring for your loved one and want to make a bigger impact to help others like them.     00;11;37;28 - 00;11;48;05  So walk me through your own thought process. What did you see the need was and how did you first go about exploring what role you could play in it beyond Maddie?     00;11;48;15 - 00;12;17;11  Yeah. So, you know, when Maddie was first diagnosed, I, you know, it took me almost a year really, to be strong enough to do more than just survive. To be honest, My, my, my, my marriage failed, and I actually became a single mum, which is not uncommon for patients or families that have children with with chronic conditions. But I did have a good job and a great family and friends who were there for me.     00;12;17;11 - 00;12;47;18  So when Maddie was diagnosed with type three, Go Shay's Disease, it's obviously have has neurological involvement. There was literally no information out there for me as a parent. And when I went to the library, it said Death within a year. And nobody had ever heard about it. So for me, you know, I set out to develop information for patients and parents so that they wouldn't be in that situation.     00;12;47;28 - 00;13;16;19  But also I set out to sort of develop and build a community in the UK, and that was really for support, for support for me, support for Maddie and support for others. Now, around five years before Maddie was diagnosed, that was the UK and Shay's Association was actually set up. Now it was set up because of this new medicine that had come up for type one Gaucher disease.     00;13;17;12 - 00;13;41;20  And as patients were going to the hospital and having treatment, they were talking to each other. And this organization set up. So there was already an established group in the UK and I decided to join them and then they invited me to sit on the board as a sort of representative for patients with type two and Type three.     00;13;42;09 - 00;14;04;10  And they asked me to do that really, because everybody else on the board had type one Gaucher disease. And if you're a patient or caregiver with a with type two or type three, it really is it's almost like a completely different disease. So I think they saw the benefits of me having the benefits of of having me on the on the board.     00;14;04;10 - 00;14;31;16  I did have a lot of support from the founders of the UK Association, Susan Lewis and Jeremy Emmanuel, and also Maddie's consultant doctor Elodie, who was really great in terms of educating me about the disease, what was going on in research, who the the doctors were, where the other patients were. So it was really a sort of collaborative effort.     00;14;32;13 - 00;15;02;29  And, you know, I started to bring patients and parents together on family days out and conferences and sort of listened to the challenges. And then we write books on education, trying to find out what was going on in research, the developments, and really how best to go about sort of trying to improve patient outcomes, whatever that looks like, to to to to a patient.     00;15;02;29 - 00;15;27;05  You know, were there any new treatments? Was there ever going to be a cure? And it was really about putting information, you know, putting my feelers out there, getting known, getting people to talk to me and, you know, feeding all that back to to the community. I became a board member of the UK Association in 2005, and I started working for them then.     00;15;27;25 - 00;16;03;01  And actually I remained working in the UK as well as sort of then going into the European and global state until about 2018. And in terms of my work, European and internationally, again before my time actually back in 1994, again because of this new treatment, seven patient advocates for this disease invited themselves to a European meeting where doctors and researchers were talking about this disease.     00;16;03;21 - 00;16;31;07  And these advocates sort of formed an anarchy of of patient of a patient group, because they you know, they saw they had common interests and goals. And by working together, they could see that they would have a much, much stronger voice. And that European sort of group of patients soon turned into a sort of international group of patients.     00;16;31;07 - 00;16;57;11  And today that's nine is the international alliance. We'll be celebrating 30 years next year. And I am the CEO of the International Gaucher Alliance and have been involved, you know, since 2008. Really, I sort of got my foot in the door in the UK and then slowly learned a lot and then sort of started to get my foot in the door in Europe and internationally.     00;16;57;29 - 00;17;24;24  But I think when I when I really think about why I did what I did and why I became a patient advocate, it really does go back to Maddie being born in the UK, you know, and she had access to treatment and good clinical care. And to me I wanted to try and make sure that wherever other patients lived in the world, that they too could have this.     00;17;25;12 - 00;18;02;06  And because treatments were so successful for many patients that, you know, there was a hope for those patients to have a future, but also that they didn't feel alone. Having a rare disease can be very lonely. And for many patients that I work with, I will never, ever meet them. But they know that there is somebody out there who's advocating on their behalf and the if they're feeling down or helpless and have nowhere to go in their own community, then actually there is somebody who does care.     00;18;02;28 - 00;18;31;19  Yeah, it is a tremendous resource and sorely needed, not just for Gaucher disease but for others. Actually, the challenges faced by those battling Gaucher disease are so similar to those overall who have or are caregiving. For someone who has a rare disease, as you just touched on it a little bit, but talk to me about what it's like to live in that world where you have something very serious, but because it's rare, you kind of feel emphasis isn't being placed on it.     00;18;31;28 - 00;18;35;00  And it can it can feel quite isolating, right?     00;18;35;08 - 00;19;00;15  Yeah. So I think I would start by saying people say you've got what I've never heard of. What is it? What is it? And, you know, this is the reality for patients and their and their caregivers, because you absolutely have to become your own advocate or the advocate of your your child. And you have to fight for everything.     00;19;00;24 - 00;19;29;01  And every time you go for an appointment, you have to again, go through what it is, how it affects. And then they're interested. You are an interesting case and that in itself is is is very, very challenging. And I think, you know, this is why patient organizations are so important because they provide the support that patients need, that, you know, that pastoral or support that time.     00;19;29;01 - 00;19;58;11  Somebody to talk to who knows how you feel and has often been through that situation. But they also provide information and advice so that they can empower you and patients and, you know, for better outcomes. But, you know, I rare diseases don't have the coverage of of more common diseases. And you may live in a country where they're just really on any other patients.     00;19;58;11 - 00;20;22;18  Speaker 2  So it can be really, really lonely. I went to Ireland many years ago with a member of our board from the UK and we actually have met a couple of patients now in Ireland and there was a gentleman there and he was in his fifties, so he'd had chase disease for 30 odd years and he had never met another patient.     00;20;23;05 - 00;20;47;25  Wow. You know, that is in a place like Ireland. So you can see how how lonely and isolating it can be not only for the patient but also for their family. I think you hear when we think about, you know, the environment we live in now, you know, social media like Facebook can be very important. Linking patients and families.     00;20;47;25 - 00;21;18;05  And often they become, you know, online communities sharing stories and advice. And we see that a lot in negotiate community. You know, type three that my daughter has many years ago there was one type three patient in in Lithuania. So, you know, by using things like social media that that parent could come into a community and sort of be part of a wider family.     00;21;18;21 - 00;21;39;27  One of the biggest things that affected us many other patients is that, you know, because it is a rare disease and people don't know a lot about it, it means that people have an unknown future. You know, when Mary was diagnosed, we read death. We knew within a year and we were told not to make any future plans.     00;21;40;14 - 00;22;10;10  Parents are told to take their children home and just enjoy life. You know, it's a physical and real emotional rollercoaster. And the thing is, you can't get out of it. It's exhausting. It's not something you can, you know, you can sort of just put to one side. It's part of everything you do. And the thing is, is reality is, is that if you don't fight as a family, nobody will fight for you.     00;22;11;01 - 00;22;57;05  And I think like many rare diseases, you know, mental health is a massive challenge for patient and caregiver community. And, you know, we are seeing much more attention being given to this topic, which is good. But like anything in life, it's patchy and not available to all. So importantly, you know, I see my role as sort of being that people often spend a lot of time, you know, just WhatsApp, a new families or people sending little videos and just having a chat and that enables me to sort of hopefully help them make them not feel so alone, but also enables me to sort of link them up with other people that I might know that, you know, they may get support from.     00;22;59;25 - 00;23;28;06  Yeah, it sounds like what families need most is connection information and ongoing research. But have you found that the perception isn't necessarily true, that there are actually medical practitioners who are working on and who are captivated by finding solutions to rare diseases? Because that sounds like what you've found. And specifically, have we gotten anywhere in terms of advanced treatments since the early nineties?     00;23;28;23 - 00;23;29;01  So. You know, I think, you know, we are really lucky and go chase disease. You know, we we work in an environment where, you know, our doctors and researchers are really committed to improve patient lives and together as a community, you know, physicians and patients, we've really developed a great global community. You've got the international Law Alliance, which is an umbrella organization, and we have 58 member countries, plus we work in another 25 or 30 countries with patients.     00;24;06;13 - 00;24;46;09  And then you have the International Working Group on Disease, which is a sort of platform for clinicians and research is globally who are interested in in Gaucher disease. And actually we do a lot of work together on things like guidelines, consensuses, meetings, and we often body doctors are who are new in go chase disease with, you know, people from the Iwg day, for instance, there was a doctor in Kenya who'd got a new patients, had never treated a patient before.     00;24;46;15 - 00;25;19;27  So we were able to, you know, buddy them up with somebody from the UK who had a lot of experience. We there is a lack of like diagnostic testing in Zambia. So we know doctors in Brazil that would do testing free of charge for patients. So we link those those doctors up and you know, there's we're working a lot in Africa at the moment and there's a lot of education to be done.     00;25;20;07 - 00;25;58;00  So we recently did a online educational session which actually got 1200 doctors from Kenya and the surrounding countries that are interested in pediatric medicine. And one of our doctors from the Iwg today did that educational session for us. So there's a lot of of volunteering, there's a lot of joint working, there's a lot of preceptorship. And, you know, it's really a great collaborative environment now, you know, that's 30 years in the making.     00;25;58;16 - 00;26;30;25  But, you know, over the last few years we've seen a real acceleration, you know, in reaching areas of the world that maybe before were cut off. You know, we didn't know that there were patients there. So that's all really positive. And, you know, we patients disease is a is a bit of a success story really in some ways because for type one patients, enzyme replacement therapy has just completely changed the patient, a patient's life.     00;26;31;13 - 00;27;04;14  You know, they have been able to get access to treatment. They have been able to get access to, you know, doctors that are very knowledgeable. And, you know, they've been able to get on with their lives, to have families, to have careers, to run marathons. And, you know, enzyme replacement therapy that came on over 30 years ago has been followed for type one Déchets disease, an oral therapy called substrate reduction therapy.     00;27;04;14 - 00;27;35;16  So we've gone from, you know, weekly or fortnightly infusions to taking a pill once or twice a day, which has transformed patients lives. And actually at the moment where we are again mostly for type one, there are a number of clinical trials for gene therapy for type one. So, you know, we've gone the real sort of like infusion pill and now we're potentially looking at a one off treatment for Gay Shea's disease.     00;27;35;25 - 00;28;07;06  Now most of that is for type one. There is currently one study for type two and one study for type three. So, you know, that's an area still where we are trying to find out how to address the neurological involvement in patients. And there's still a long way to go to go for that. I think the other thing I would say is that one of the challenges for us as a global community is that, yes, we are 30 years down the road from that first enzyme replacement therapy.     00;28;07;19 - 00;29;01;05  But if you are born in Kenya or Zambia or Cuba or Pakistan or Jordan, treatment is not available through reimbursement. So that is where there is still a lot of work to do. Now, there are opportunities for patients to get treatments, and that's through charitable access programs. And that's one of the things that we as an organization, the International Gateway Alliance, do a lot with the companies who manufacture these therapies is that we work with them and they have a number of slots for charitable access where patients can be given treatment, a lifelong treatment where there's no river reimbursement in their countries so that they can have that future and that life.     00;29;01;05 - 00;29;25;05  You know, we've talked to guests several times on this podcast about patient centered research and citizen science. And it feels like, especially with rare diseases, there needs to be a tighter collaboration where patients and their families are more involved and work more closely and directly with researchers. Are you seeing that happening and is the research side leaning into it?     00;29;25;16 - 00;30;04;07  So I think the this is an area that we have been really strong on in as I described earlier, you have the patient community and you have the clinical community who have sort of grown up together and are working very, very closely together. And then you've got the research community and the sort of pharma community who, you know, are not big pharma, they are small pharma, and they recognize that patient centered research and and patient centered support needs to run through everything they do.     00;30;04;23 - 00;30;36;06  And what we see is that we see that we are invited to have a seat at the table from very, very early on to really understand the condition, to understand the challenges that patients and their families face, to understand what's important to patients and what areas they're still very little known about. And we can we deliver that to the different stakeholders.     00;30;36;21 - 00;31;17;10  And basically we are seen as an equal partner and, you know, things like the development of research projects, the development of clinical trial design, the patient community are really co-creators in this. I think this is an area where we have had a lot of success. So I think generally in rare diseases that you see that there is a real recognition of the value of having patients and patient advocates at the table from the very beginning, because ultimately whatever you're doing, you want it to go as smoothly as possible and you want it to have the biggest effect.     00;31;17;22 - 00;31;39;11  Now, if you get everybody around the table at the very beginning, you're more likely to see that happen. Whereas if you get to a point and then you say, Oh, actually maybe we should invite a few patients or patient caregivers or advocates around, see what they think, and then you're having to go back or you're not actually doing that.     00;31;39;24 - 00;32;07;23  And then I think, you know, we're seeing more encouragement and necessity by health technology assessments and marketing for licensing like the FDA and EMA to say, you know, where is your involvement from your patient community? How did you know your patient? How did you work with your patient community? What is important to patients? What do they want you to address?     00;32;07;23 - 00;32;16;03  Well, in fact, I think you've partnered with Cerner and Visa, which is now part of Oracle Life Sciences, for some research efforts. What does that partnership look like?     00;32;16;21 - 00;32;43;08  Yeah, so this is really, really exciting. So as I've said, you know, a lot of work in progress has been done for patients with type one diabetes disease, not so much for type two and Type three. And I've been in this community for 26 years and I've sadly seen many of our patients lose their lives to type two and type ricochets disease now because it is very rare.     00;32;43;27 - 00;33;20;12  No single center has enough patients to study. There is a real lack of natural history data on these patients. And actually when you look at patients, even when they have like the same genotype, they have completely different phenotypes. You know, they the way that the disease presents itself in them is completely different. And therefore, it's a really, really challenging disease to really understand and see how you could potentially develop a therapy that is safe and effective.     00;33;20;21 - 00;33;54;03  But also, you know, how should we be managing these patients clinically and what are the care and support do these patients need to function in society? So what we did as an international alliance is that we had an idea to set up a patient led patient owned registry just for type two and three disease. And for the last couple of years, we have been working with what was Sunrun Visa and now Oracle, in setting up a registry called Guardian.     00;33;54;13 - 00;34;27;07  And Guardian collects patient reported data from patients and caregivers around the world on the way that Type two and Type three patients disease affect patients in their everyday life. And the way that we set it up was we work collaboratively with interviewing patients, doing focus groups, finding out what was important to them, and then developing the question, as in Guardian and the role of Sutter and Visa.     00;34;27;07 - 00;34;53;26  Oracle is that they are the they are the the organization that provide all of that support to us in terms of running the registry as a patient organization. We had a vision, we had an idea, we had the passion, but we didn't have the skills and resources in in-house to run a registry. And that is where Sutter and Visa Oracle have come in and we've developed this partnership.     00;34;54;11 - 00;35;14;21  Well. So on the research side, obviously clinical trials are where the rubber meets the road. If I have a rare disease, is it getting easier for me to participate and find a clinical trial if I want to? And how easy is it now for researchers to find the patients willing to participate in these trials?     00;35;14;21 - 00;35;48;13  So so we do have some trials for go shows disease, but because it's a rare disease, it's not like working in, you know, oncology or diabetes or anything like that. So, you know, there are not a lot of trials, but there are trials. And, you know, the thing is, is that we have a lot of centers of excellence around where patients go to have their disease managed.     00;35;49;01 - 00;36;35;17  So the this is an ideal opportunity where you have cohorts of patients to be able to raise awareness and make patients aware that, you know, there are clinical trials for their type of disease. I've got to say the one of the challenges is that most clinical trials are done in the West, whereas we have huge unmet need in the east of the world and that comes down to expertise, but it also comes down to it's easier to run a clinical trial in Europe than it is in Africa, which is something that we as a patient organization are trying to work with.     00;36;36;00 - 00;37;22;22  Those interested in bringing clinical trials to shades disease to try and have a different approach. And I think because we have the International Working Group on disease and we have a very strong patient organization and we have a few really good pharma companies that are interested in Roche's disease, We, you know, we work together. So I think that it's not easy to develop trials, but but actually it's also not easy to recruit for clinical trials because patients have also had enzyme replacement therapy or substrate reduction therapy for a number of years now.     00;37;22;22 - 00;37;53;04  And these drugs have been really, really good. They do what they say on the tin and patients are living a really good quality of life. So actually recruiting patients into these clinical trials can be quite challenging, particularly when you're thinking about new technologies and patients want to know about safety and efficacy and the benefits of switching from a a safe therapy, which they've been on for for years into something that is experimental.     00;37;53;19 - 00;38;08;22  So as an organization, you know, we are really trying to raise awareness and share information on clinical trials and educate patients on clinical trials, you know, to support their decision making.     00;38;09;12 - 00;38;33;11  Well, Tanni, it's been so great hearing your story, hearing Maddie's story. There are real people and human beings behind these rare diseases, and you've done such great work bringing that vibe to health care and the work being done on pragmatic solutions. I'm going to bet that listeners will want to learn more about you and what you're doing. Is there any way they can get more information or get in touch with you and the Alliance?     00;38;34;00 - 00;39;03;17  Yeah. So like most organizations, we have a great website which is Geisha Airlines dot org and we can also be found on social media platforms like Facebook and other sorts of Instagram. So we, you know, we do have a really good social media presence for you to do is is is find out where the newsletter is or click on the join us on on Facebook.     00;39;03;17 - 00;39;13;27  And you know, we have somebody in our group that works specifically on communication and we try and share not only the work we do, but work that our partners do.     00;39;14;06 - 00;39;47;16  Perfect. Well, thank you so much again. And if you faithful listener want to find out how Oracle can simplify and accelerate your life sciences research, just check out Oracle dot com slash Life Sciences. 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