Terry Pirovolakis News Today

Ascendis, Bayer, Boehringer Ingelheim, Jazz Pharmaceuticals, Novo Nordisk, Sanofi, and Teva to keynote sharing innovations to drive better patient outcomes BARCELONA, Spain, May 21, 2024

When a disorder affects only a few people around the world, companies see little incentive to find out what could treat it. Increasingly, advocacy networks are funding and promoting their own research

Trial participants with Spastic Paraplegia 50 (SPG50) and Charcot-Marie-Tooth disease type 4J (CMT4J) could benefit from this joint effort to manufacture...

The new project, Bespoke Gene Therapy Consortium, hopes its work on 8 rare diseases will set the standard and framework to help thousands of others.

The new project, Bespoke Gene Therapy Consortium, hopes its work on 8 rare diseases will set the standard and framework to help thousands of others.

The new project, Bespoke Gene Therapy Consortium, hopes its work on 8 rare diseases will set the standard and framework to help thousands of others.

Gene therapy for ultra-rare diseases afflicting children stand to significantly advance with a ground-breaking launch of Social Purpose Corporation, Elpida Tx.

A Toronto family’s heart-wrenching journey to find a cure for their son’s ultra-rare disease has reached a new milestone.

After a Toronto boy became the first person to receive a new gene therapy to treat the rare genetic disease spastic paraplegia type 50 or SPG50 earlier this year, his family hopes to connect sick children around the world with the treatment too.

Michael Pirovolakis, a four-year-old boy in Toronto, is now the only child in the world to receive a potential cure for his extremely rare disease. After his parents desperately raised $3 million over a period of three years, they finally have faith for their son’s possible recovery. He is the on