News Medical

UCLA researchers reveal reversibility of Friedreich's ataxia in mouse models

In children and adults with Friedreich's ataxia, an inherited disease that causes damage to the nervous system, a loss of coordination typically progresses to muscle weakness and can involve vision, ...
The American Journal of Managed Care

Engaging mFARS Scale to Monitor Friedreich Ataxia Progression

An expert in Friedreich's ataxia evaluates the mFARS (modified Friedreich's Ataxia Rating Scale) measurement tool, highlighting its advantages and limitations, while also exploring alternative ...
Healio

FDA requests more studies before approving vatiquinone for Friedreich’s ataxia

Please provide your email address to receive an email when new articles are posted on . Vatiquinone inhibits 15-Lipoxygenase, which regulates the pathways that Friedreich’s ataxia disrupts. PTC ...
EurekAlert!

University of Oklahoma researcher makes breakthrough discovery about Friedreich’s ataxia genetic defect

OKLAHOMA CITY – A University of Oklahoma researcher is the first to discover that the sequence of the genetic defect in the neuromuscular disease Friedreich’s ataxia isn’t always as uniform as ...
Business Wire

Papillon Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia

SAN DIEGO--(BUSINESS WIRE)--Papillon Therapeutics Inc., a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited ...
Healio

Q&A: Advocates meet with lawmakers in support of patients with Friedreich’s ataxia

Please provide your email address to receive an email when new articles are posted on . Approximately 5,000 people in the United States have Friedreich’s ataxia. Advocates virtually met with ...
FierceBiotech

PTC puts Friedreich ataxia drug's past failures in the rearview with long-term data drop

Several months after forging a fresh path forward for its troubled Friedreich ataxia (FA) drug vatiquinone, PTC Therapeutics is back with some much-needed positive data. The candidate passed muster by ...
FierceBiotech

FDA rejection derails PTC Therapeutics' rare disease hopeful

PTC Therapeutics has long been trying to workshop its vatiquinone into a marketable treatment for Friedreich's ataxia (FA) after a phase 3 fail in 2023. Despite its efforts, the FDA has snubbed the ...
The American Journal of Managed Care

Managing Adverse Events in Novel Friedreich’s Ataxia Therapy

A key opinion leader outlines common adverse events associated with omaveloxolone treatment in Friedreich's ataxia patients and describes strategies used to manage or minimize these adverse effects.
Hosted on MSN

Biogen Launches Phase 3 Pediatric Trial Of Omaveloxolone For Friedreich Ataxia.

Biogen Inc. (NASDAQ:BIIB) is among the 11 Best Genomics Stocks to Buy According to Hedge Funds. It has begun dosing in the global Phase 3 BRAVE trial to test omaveloxolone (SKYCLARYS®) in children ...
Hosted on MSN

Biogen’s Friedreich’s ataxia treatment authorised in UK

Biogen Netherlands has received marketing authorisation from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for its Skyclarys (omaveloxolone) to treat Friedreich's ataxia in ...