Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Of course, the health risks of too much sunlight are well-known and include skin cancer, immune system suppression, and vision-damaging eye conditions like cataracts and macular degeneration. The key ...

Despite modern high-throughput sequencing, the genetic cause of most rare movement disorders remains unclear. A research team in Bochum and Tübingen has now solved one piece of the puzzle: The ...

There is a process to a patient being diagnosed with a rare condition and then collaborating with their physician, if they are fortunate enough to have that opporunity. One recent qualitative study ...

Atamyo Therapeutics, a biotechnology company specializing in the development of next-generation gene therapies for limb-girdle muscular dystrophy (LGMD), announced at the MDA (Muscular Dystrophy ...

The company’s positioning frequently appears alongside broader movements.

New analyses out to 24-months showed improvement in heart and lung function compared to expected declines in DMD natural history -- Data expand ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced presentations on SGT-003, its ...

Sildenafil – an active ingredient also marketed under the name of Viagra – improves symptoms in patients with Leigh syndrome. This has now been reported in the Cell* journal by researchers at Charité ...

Brooklyn Nets radio play-by-play voice Chris Carrino Sr. was honored by the Naismith Basketball Hall of Fame with a Curt Gowdy Media Award.

Vinay Prasad’s exit — his second from the agency in less than a year — comes after sharp criticism of his handling of drug ...

Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa" or the "Company"), a clinical-stage biopharmaceutical company developing novel therapies in oncology and other rare diseases, today announces the ...