Indo US Organization for Rare Diseases (IndoUSrare)的动态

Indo US Organization for Rare Diseases (IndoUSrare)

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Dr. Abbey Meyers, Founder of National Organization for Rare Disorders, shared a heartfelt message honoring the Abbey Meyers Khushi Award recipients: Dr. Rahul Purwar (ImmunoACT), Dr. Hasmukh Jain (Tata Memorial Centre), and Frank Sasinowski (Hyman, Phelps & McNamara, P.C.) at the Indo US Bridging Rare Summit 2024. Relive this special moment through this video as we celebrate their exceptional contributions to rare diseases. ?? Centre for Cellular and Molecular Biology BRIC-Centre for DNA Fingerprinting and Diagnostics (CDFD) AIIMS (All India Institute of Medical Sciences, New Delhi) Virginia Bio George Mason University BioHealth Innovation, Inc. BioHealth Capital Region Sir Ganga Ram Hospital Tamahar Trust Aarogya Seva: Global Health Volunteer Alliance Beyond the Diagnosis #SocietyforIndianAcademyofMedicalGenetics Harsha K Rajasimha Narayanan Govindarajan Reena Kartha Nisha Venugopal Ramya T karur Saundarya M S Indo US Organization for Rare Diseases (IndoUSrare) #RareDiseases #AbbeyMeyersKhushiAward #PatientAdvocacy #bridgingrare #bridge4rare #roar4rare

Transcript

I want want to congratulate you. On the gala and the award ceremonies for 2024, and I want to say that your organization, I know is thriving and attracting the admiration of many charities and support groups throughout the world. So you are appreciated and you are drawing. Attention to the lesser known and very serious diseases, the rare diseases that are generally ignored. And, umm. This year, I'm very pleased to know that one of your arteries is. An old friend of mine. Frank says it Aski, an attorney from Washington, DC. And I have no frank for about. 40 years, more or less. And I think we first became acquainted when he was working. At the FDA as a young lawyer is one of his first jobs and he was assigned a small task. Writing the regulations for the Orphan Drug Act. And since. I was known as the person who got the Orphan Drug Act through Congress. That's how we became acquainted, because he would say. Well, what did you mean by this? Whatever the words were in the. And yeah. Look, the law. Umm. So Frank and I became acquainted at that time, but primarily it was not because of his interest in the law, but because we both have sons. With a rare disease called turrets syndrome. And at the time is considered extremely rare that there was hardly and there was. I remember one of the doctors in New York who was known as one of the the most important researchers that ever looked at the disease and and he felt there wasn't even 100 patients in New York State. That had Tourette syndrome and today only because of the publicity and, and putting Tourette's syndrome in in movies and books and so forth, it's become much better known and. Even though people think about the the symptoms. As if everybody has every symptom which they don't. Uh, it has made it much more accepted disease. And, umm. After. After we met and after Frank and I knew that our we had sons with the disease. Frank devoted the rest of his career to helping pharmaceutical companies to develop new orphan drugs and to get the treatments through the FDA approval process. So that it could get to pharmacy shelves and that. As you probably know, is is the most difficult part of drug approval process. It's getting it through the regulatory agencies who have the responsibility for approving treatments that eventually can get to patients. So, umm. That brings me to the other two honorees tonight. Who? Who? Have been working together for for some time. Doctor Karwar and Doctor John Who have worked to reduce the costs. Of expensive car T therapies, which are known mostly as the treatments for rare cancers. Umm. I want to salute the work that they are doing because the high cost of some of the most unique life saving therapies. Can prevent patients from obtaining them. I read that the most recent gene therapy approved that the United States is with sickle cell anemia, which kills people. Every year people are dying of of sickle cell disease. And, umm. Uh. The cost of the gene therapy is 3 million U.S. dollars a year, 3 million a year. How many Americans can afford that? Very, very few and so. I've always been praying that someone would come along and find a less expensive way to manufacture these drugs so that they become affordable to the people who need them. Umm, so here I see that it has happened on the car T therapies that are used for rare cancers and that these doctors in India have been developing the technology that would enable them to do that. And. I want to thank Doctor Paroy and June Jane. I want to say thank you very much, not just for the patients who are going to need the CAR T cell therapies. But for all of the patients who are using new technology and nobody is looking for less expensive ways to to make them except for you. So you have a lot of work ahead. And if you ever run short of ideas on which drugs to to look for next, just let me know. Umm. So after you finish the car to you sell therapies. I invite you to specifically look at gene therapies. Because they are out of the reach of everybody and, and very often people outside the United States look at the United States and feel that we're all millionaires and we could afford anything. And the truth is no. There are large portions of a populations who are poor. There are others who are just managing to hang on, but, but. The first priority is is they're written and the food for their children, etcetera. And so there are many people in the United States who cannot afford the most advanced forms of. Of. Therapies that will. Cure or at least treat their their children and their loved ones. The most expensive treatments in the world right now are for genetic disorders, hereditary disorders that are inherited from one generation to the next. So if you or any of your colleagues could do anything to lower the costs. Of treatments that will make them more affordable all over the world. Because there are so many places where they cannot afford. $1,000,000 prices. I'm not on drugs and I, I know you will succeed at what you're doing. So thank you very much for the opportunity. Just to introduce you all. I really wish that I could be there for the, the ceremony, but Frank will tell you I'm 80 years old and next year in April. Will become a great grandmother so I like to stay home and feel safe. Thank you very much. Bye bye.

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