Celebrating Many Milestones This Rare Disease Day!

Today, I wake up with much more enthusiasm and zeal to celebrate #RareDiseaseDay with a community of 350+ million worldwide. It all began in the United States in 1983, when the Orphan Drug Act (ODA) was signed into law. This year, we are celebrating 40th anniversary of the US FDA ODA.

No better way to celebrate today than with a fireside chat with the person who sparked this revolution, Dr. Abbey S. Meyers. There are many events to balance and choose from today as RARE advocates and warriors are most active today. Please join us in this event to honor Dr. Meyers and other leaders in the US and around the world who have contributed to the neglected sufferings of families affected by rare and genetic diseases. There is still time to register for free here: https://rddindousrare2023.eventbrite.com/

I was first a computer science engineering major and then a computational biologist researcher with experience spanning NIH and FDA resulting in numerous publications involving genomics and precision medicine. Then came a day when I became a rare dad, to a child born with Edwards Syndrome in 2012. It is not an inherited disease, it is a congenital anomaly that may occur post-conception. Life got a purpose and new meaning after that. I draw inspiration from Dr. Abbey S. Meyers in the US. From Dr. Ishwar Verma in India who was recently recognized with the 4th highest civilian award for his contributions to medical genetics and rare diseases in India. We have much to celebrate today with about 1100 approved orphan drugs by the FDA but much more work to do as there remain 10,000 more diseases without any approved treatment options.

But we got hope, aspirations, determination, grit, and resources today than ever before. There are governmental policies, grants, funding, and a thriving biopharmaceutical industry that is making orphan drug development a core part of their strategy. About a third of all drugs approved by the FDA each year are orphan drugs or precision medicines. With about 2000 active clinical trials involving cell and gene therapies that have the potential to "cure" single gene disorders affecting millions worldwide such as Sickle Cell Disease, Hemophilia, Duchenne Muscular Dystrophy, and many more.

We got work to do to make sure these therapies are developed faster, cheaper, and made more accessible to patients globally. SPEED is the key - which takes a global village to collaborate on digital platforms of this generation.

I invite you to join me today at #RDDindousrare #Art4Rare contest results will be announced after the fireside chat with Dr. Meyers.

I am now heading out to exhibit and join advocates at the #RDDNIH event at the NIH campus in Bethesda, MD!

Where are you going today? Please leave your comments and questions below. Visit https://indousrare.org