An international team of scientists has modified a standardized neurological assessment so it can better measure the severity of non-movement…
Articles by Marisa Wexler, MS
SGT-212, a gene therapy candidate for Friedreich’s ataxia (FA) that’s currently in early clinical testing, has been granted rare…
International Ataxia Awareness Day is coming up this Thursday, and organizations around the world are getting ready for this annual…
Levels of mitochondrial complex 1 (MC1) — a group of proteins essential to the function of mitochondria, cells’ powerhouses —…
The U.S. Food and Drug Administration (FDA) has declined to approve the oral therapy vatiquinone for treating children and…
A committee of Canada’s Drug Agency (CDA) has recommended that insurers should cover the cost of Skyclarys (omaveloxolone)…
Voyager Therapeutics expects to begin clinical testing of its experimental gene therapy for Friedreich’s ataxia (FA) in 2026. The…
Larimar Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) this year to grant accelerated approval to…
Errors during repair when DNA gets damaged may give rise to the mutations that cause Friedreich’s ataxia, a study…
The U.S. Food and Drug Administration (FDA) has given Solid Biosciences the go-ahead to start clinical testing of…