Title: Initial Experience from the INSPIRE DUCHENNE Phase I/II Study of SGT-003 Microdystrophin Gene Therapy for Duchenne ...

Today, Benzinga 's options scanner spotted 32 uncommon options trades for Biogen. This isn't normal. The overall sentiment of these big-money traders is split between 37% bullish and 31%, bearish. Out ...

There are several current treatment options for Friedreich's ataxia, or FA as I'll call it for the rest of this time. The most important is what we had before the approval of any drugs.

As announced on December 4, 2024, Solid entered into a collaboration with Mayo Clinic to develop an AAV gene therapy platform ...

Twenty-seven kindreds with Friedreich's ataxia were seen at the Johns Hopkins Hospital from 1935 to 1957. In three kindreds the disease exhibited an apparently dominant mode of inheritance. Affected ...

Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems ...

Other symptoms include a painful bluish coloration of hands and feet (acrocyanosis or Raynaud sign), mottled discoloration of the skin (livedo reticularis), splenomegaly, respiratory problems (in CAD ...

From reproductive rights to climate change to Big Tech, The Independent is on the ground when the story is developing. Whether it's investigating the financials of Elon Musk's pro-Trump PAC or ...

Turns out a “Supernatural” reunion is happening after all — just for a completely different series. Amazon Prime Video’s hit (anti-) superhero series “The Boys” revealed Wednesday it ...

Maybe I need to talk about something [too].’” Never miss a story — sign up for PEOPLE's free daily newsletter to stay up-to-date on the best of what PEOPLE has to offer , from ...