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.

The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich ataxia (FA).

Friedreich's ataxia is a rare and life-shortening neuromuscular disorder that affects the central nervous system and the heart. With symptoms typically emerging in childhood or adolescence ...

"We are excited to be one step closer to bringing an approved therapy to all patients with Friedreich's ataxia," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics.

Have you ever wanted to learn sign language to communicate with family members, friends, or other people who are deaf? If so, you might want to try a new interactive website that uses AI to train ...

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

Pisces is the Zodiac's final sign, ruling the last days of February and a majority of March. Symbolized by the fish, Pisces is a water sign. Those born under Pisces are known for their acute ...

After getting a green light from the European Commission, Biogen’s Skyclarys is the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA) in the EU.

including Duchenne muscular dystrophy (Duchenne), Friedreich’s ataxia (FA), catecholaminergic polymorphic ventricular tachycardia (CPVT), TNNT2-mediated dilated cardiomyopathy, BAG3-mediated ...

It also markets SPINRAZA to treat SMA, SKYCLARYS for the treatment of Friedreich’s Ataxia, and other drugs. The company is focusing on the four products it launched last year for Alzheimer’s ...

LEXINGTON, Mass., March 11, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company ...

Biogen’s Skyclarys is on course to becoming the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA) in the EU after it was recommended by the EMA’s human ...