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Fragile X disorders include two distinct conditions: fragile X syndrome (the most common single-gene cause of developmental ...
In this video, Khalaf Bushara, MD, FRCP, discusses therapies in the pipeline that show promise for the treatment of Friedriech's ataxia.
Michael Gehr, of Edmond, and his family are on a mission to help find a cure for a rare genetic disease that he was diagnosed with at age 15.
Curtains up for a Cure: The Gehr family’s fight against Friedreich's Ataxia Local family host soiree to raise money and awareness about Friedreich Ataxia. Friday, June 6th 2025, 10:09 pm ...
Huntington’s disease and Friedreich’s ataxia are part of a group of neurological disorders dubbed trinucleotide repeat disorders because they are caused by repeating three-letter stretches of DNA.
Quince Therapeutics advances Ataxia-Telangiectasia treatment with innovative AIDE platform, insider support, and NEAT Phase 3 trial data expected early 2026.
Ataxia is caused by damage in cerebellum, leading to poor muscle control. Know the causes and types of ataxia that can lead to other health complications.
The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for vatiquinone for the treatment of children and ...
About Fast Track Designation The FDA’s Fast Track program facilitates the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and ...
In some cases, an inability to balance is referred to as a symptom known as ataxia. But ataxia can also be its own condition or disease.
Bill Nye, one of the nation's most popular scientists is headed to Capitol Hill to bring awareness to Ataxia.
Ataxia, often misdiagnosed as Parkinson's or MS, has affected Bill Nye's family — and now he's spreading the word on genetic testing.