RTÉ Ireland

'Our lives are worth saving' - Friedreich's Ataxia patients in call for drug

People living with a rare neurological degenerative disease have called on the Government to make the first treatment for the ...
Fierce Biotech

'Like talking to a brick wall': Senate hearing takes aim at FDA's rare disease review process

A panel of physicians, biotech leaders and patient advocates took aim at the FDA during a Senate hearing Thursday on how the ...
MarketWatch

Friedreich's Ataxia Market Poised for Rapid Expansion During the Forecast Period (2025-2034) as Drug Pipeline Gains Momentum | DelveInsight

LAS VEGAS, June 5, 2025 /PRNewswire/ -- DelveInsight's Friedreich's Ataxia Market Insights report includes a comprehensive understanding of current treatment practices, Friedreich's ataxia emerging ...
Seeking Alpha

Larimar started at buy by Truist on Friedreich's ataxia drug

Truist has initiated coverage of Larimar Therapeutics (NASDAQ:LRMR) with a buy rating, citing an "asymmetric" risk/reward ahead of regulatory updates for its drug candidate nomla for Friedreich’s ...
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 ...
Stocktwits on MSN

Why did Larimar Therapeutics’ stock surge over 30% pre-market today?

The therapy is to treat adults and children with Friedreich’s ataxia, a progressive neurodegenerative disorder. ・Preliminary findings from the open-label study show that nomlabofusp increased FXN ...
Nasdaq

Biohaven Announces FDA Acceptance and Priority Review of Troriluzole New Drug Application for the Treatment of Spinocerebellar Ataxia

Spinocerebellar Ataxia (SCA) is a rare, genetic, life-threatening neurodegenerative disease with no available treatment. Troriluzole demonstrated a 50-70% slowing of SCA disease progression on the ...
BioWorld

FDA rejects PTC’s Friedreich’s ataxia drug, for now

It looks like Biogen Inc.’s Nrf2 activator, Skyclarys (omaveloxolone), will maintain its status as the sole therapy approved for treating patients with Friedreich’s ataxia (FA), at least for now. The ...
Business Insider

Solid Biosciences Receives FDA Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

- SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations - - Dosing of the first participant in the Phase 1b FALCON trial has been completed, with initial data ...
Seeking Alpha

FDA Issues Complete Response Letter for Biohaven's VYGLXIA (troriluzole) New Drug Application for Spinocerebellar Ataxia

The troriluzole clinical development program encompassed the first industry clinical trials to generate data showing therapeutic potential in patients with spinocerebellar ataxia (SCA), a rare genetic ...
Morningstar

FDA Rejects PTC Therapeutics Friedreich's Ataxia Drug

The U.S. Food and Drug Administration has turned away PTC Therapeutics' proposed vatiquinone treatment for the rare, inherited neurodegenerative disorder Friedreich's ataxia over concerns about the ...