Friedreich's Ataxia

Larimar Therapeutics Inc (NASDAQ: LRMR) has received feedback from the FDA regarding the clinical hold on its CTI-1601 program.  The FDA stated it is maintaining its clinical hold and that additional data is needed to resolve the clinical hold.  Larimar is further...

The FDA has instituted a clinical hold on Larimar Therapeutics Inc's (NASDAQ: LRMR) CTI-1601 clinical program as a treatment for Friedreich's Ataxia (FA), a neurodegenerative movement disorder. The clinical hold follows the previous notification by Larimar to the FDA of...

Larimar Therapeutics Inc (NASDAQ: LRMR) shares are trading lower despite announcing positive topline data from its Phase 1 multiple ascending dose trial evaluating CTI-1601 as a treatment for Friedreich's Ataxia (FA). The data showed that repeated subcutaneous injections of...

Larimar Therapeutics Inc (NASDAQ: LRMR) has announced topline data from its Phase 1 multiple ascending dose trial evaluating CTI-1601 as a treatment for Friedreich's Ataxia (FA). FA is a genetic, progressive, neurodegenerative movement disorder with a typical onset between 10 and 15...

Horizon Pharma PLC (NASDAQ: HZNP), a biopharmaceutical company that focuses on unmet medical needs, announced Thursday topline results from a Phase 3 study called STEADFAST. The trial evaluated Horizon's ACTIMMUNE therapy for the treatment of of Friedreich's ataxia, a rare inherited...

Goldman Sachs started coverage of Horizon Pharma PLC (NASDAQ: HZNP) with a Buy rating and $24 target price, implying 34 percent upside, on the strength of its emerging orphan franchise. 'Attractive' Story "We view HZNP as an attractive SOTP story in transformation with management using the strong...