President Donald Trump’s nominee for FDA Commissioner, Martin A. Makary, MD, sought to assure senators that if confirmed, he would support efforts to expedite reviews of new drugs indicted for rare ...

With a U.S. green light for its encapsulated cell therapy technology in the bag, privately held biotech Neurotech ...

Ophthalmologist Hakam Ghabra shares what every parent needs to know about this rare eye disease and what to do to prevent ...

Still, Novo is steadfast in its goal to eventually become a leader in the space, the company’s EVP of rare disease, Ludovic ...

March 7, 2025 – The FDA has approved a new gene therapy for macular telangiectasia type 2 (MacTel), a rare eye disease that causes vision loss. The approval marks the first and only ...

Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more ...

Board members Dr. Laura Landerman Garber and Nancy Biederman update the work of NH's council, legislation and an upcoming ...

Alnylam has said it will not proceed with a clinical trial of its RNAi drug vutrisiran in the rare eye disorder Stargardt disease – at least for now – tying its decision to President Joe Biden ...

Rare POLG mitochondrial disease explained after Prince Frederik of Luxembourg died aged 22 following his diagnosis when he ...

Cherokee Nation Principal Chief Chuck Hoskin Jr signed an executive order in February 2024 establishing a Rare Disease Task ...

Rare diseases aren’t as rare as the appellation suggests. For instance, according to the US Government Accountability Office, 30 million Americans have a rare disease, a little less than 10% of ...