Health Canada has granted approval for Biogen’s Skyclarys (omaveloxolone) to treat Friedreich’s ataxia (FA), a rare, progressive neurodegenerative disease, in individuals aged 16 years and above. The ...

Solid Biosciences Inc.’s SLDB share price has dipped by 14.97%, which has investors questioning if this is right time to buy.

In addition, I think the recognition across all diseases where not everyone is going to be an expert in FA, and people can refer readily to tertiary care centers and even ordinary care centers that ...

In this video David Lynch, MD, PhD, discusses the effect Friedreich's ataxia can have on patients' quality of life.

Barry Byrne, MD, PhD, Powell Gene Therapy Center at the University of Florida, discusses gene therapy considerations for pediatric patients and how newborn screening can influence outcomes for ...

Dunlavin was alive with the sounds of music and song when the community came together at St Nicholas Church of Ireland for a ...

Anthony Monaco was a very active teen—involved in soccer, wrestling, and even cross country—but he always felt a little awkward physically. It started with running a little differently than the kids ...

Larimar Therapeutics, Inc.’s LRMR share price has dipped by 10.24%, which has investors questioning if this is right time to ...

PTC's diversified pipeline, including vatiquinone for Friedreich's ataxia, could significantly enhance revenue if approved, targeting a $2.8 billion market. Financial risks remain due to reliance ...

“Friedreich’s Ataxia is a genetic condition inherited from both parents, much in the same way as cystic fibrosis,” she began. “Neither of the parents may have the condition, but their ...