SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations - - Dosing of the first participant ...

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 ...

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 ...

Quince Therapeutics (QNCX) rated Buy ahead of Phase 3 NEAT readout in A-T; eDSP targets $1B+ orphan market with strong cash ...

Biogen Inc. (NASDAQ:BIIB) is one of the best S&P 500 stocks with huge upside potential. As of June 16, Biogen emerged as a prominent contender at the Cannes Lions International Festival of Creativity ...

The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for ...

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 ...

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 ...

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 ...

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 ...