Swathi, the Pompe Warrior

On 15th April 2021, the Annual International Pompe Day , Siva SK from Malaysia shares an awareness video about his daughter. This year’s theme is “Pompe Around the World,” the International Pompe Association (IPA) announced. The event’s slogan is “Together We Are Strong.” ITS NOT HOW YOU FALL, IT’S HOW Read more…

OpenTreatments Foundation Launches Software to Decentralize and Accelerate Drug Development for Rare Genetic Diseases

OpenTreatments Foundation will enable treatments for genetic diseases regardless of rarity or geography using an open source software platform; Sanath Kumar Ramesh built the project to address his son’s rare disease, now that work will be available to all. Sanath Kumar Ramesh – Founder & CEO of OpenTreatments Foundation Sanath Read more…

Data ownership as leverage in accelerating rare disease drug development? Finally, there’s a cloud software that empowers patient groups!

Patient groups can optimize patient registries, cross-sectional studies, and/or longitudinal natural history studies to maximize collaborations with sponsors of orphan drugs and gene therapies A disease is considered rare if it affects fewer than 200,000 people in the United States. There are over 8,000 named rare diseases and each year, Read more…

Avoid These 5 Real-World Data Pitfalls When Planning your Patient Registry, Natural History, or Other Clinical Study for a Rare Disease

Author: Harsha K Rajasimha, MS, Ph.D. Editor: Sharlene Brown, Ph.D. When clinical investigators and study coordinators start planning a new study design, speed and efficiency are paramount. Running a clinical study takes careful planning. Whether this is your 1st clinical study or your 21st, the last thing you probably want Read more…

Social entrepreneur on a mission to bridge the clinical research divide between the USA and India with advocacy and technology

Dr.Harsha Rajasimha is a rare disease social entrepreneur globally recognized for his work in genomics data science, persistent advocacy, and technology innovations addressing grand challenges facing the estimated 350 Million people suffering from rare diseases world-wide. Harsha earned his bachelor’s degree in computer science and engineering from Bangalore University, a Read more…

Sukanya

My name is Graves’ Disease…

Sukanya who faces  Rare Conditions called Graves’ Disease and Euthyroid Eye Disease (TED) sends in a poem which describes her health issues Sukanya’s article was earlier featured on Possiabilities This is poem about my disease…..Sukanya Das Hi. My name is Graves’Disease… JUST REMEMBER NOT EVERYONE GETS EVERY SYMPTOM!!!!!! I’m an invisible autoimmunedisease Read more…