Biogen Canada Inc. is disappointed by INESSS's recommendation against listing SKYCLARYS™ (omaveloxolone), despite its therapeutic potential for individuals living with Friedreich ataxia (FA) — a ...

Initial Data from the Ongoing, Blinded Phase 1 Single-Ascending Dose Trial Has Demonstrated Favorable Safety and Pharmacokinetics IND Application Submitted to Expand RESTORE-FA Trial to U.S. Sites; ...

Researchers have developed a gene editing strategy that can repair the genetic problems that lead to Huntington's disease and Friedreich's ataxia. | Genetics And Genomics ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

The global Oligonucleotide Synthesis Market, valued at US$8.9 billion in 2024 stood at US$10.5 billion in 2025 and is projected to advance at a resilient CAGR of 18.6% from 2025 to 2030, culminating ...

Biogen’s Skyclarys is on course to becoming the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA ... backed the drug as a treatment for FA in patients ...

The Delaware Division of Small Business announced the winners of the Spring 2025 Encouraging Development, Growth and ...

Voyager struggles with a negative enterprise value, financial risks, and limited catalysts in a competitive Alzheimer's ...

Its lead product candidate is CTI-1601, which is in Phase 2 OLE clinical trial for the treatment of Friedreich’s ataxia, a rare, progressive and fatal genetic disease. The company is based in ...

We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, ...