Plano-based Reata Pharmaceuticals announced Tuesday that Skyclarys, the first Food and Drug Administration-approved treatment for the ultra-rare neuromuscular disease Friedreich's ataxia, is now ...

The dynamics of the Friedreich's ataxia market are anticipated to change as companies across the globe are thoroughly working toward the development of new drug therapy options to treat this disease.

Levacetylleucine (Aqneursa) improved neurologic symptoms and functioning in adults and children with ataxia-telangiectasia in ...

LONDON--(BUSINESS WIRE)--Technavio has announced their latest drug pipeline analysis report on spinocerebellar ataxia. The report includes a detailed analysis of the pipeline molecules under ...

Friedrich’s ataxia, an ultra-rare disease characterized by progressively worsening muscle function that ultimately leads to death, now has its first FDA-approved therapy. The regulator has given the ...

Wedbush has cut its rating of Design Therapeutics (NASDAQ:DSGN) to neutral, stating that the company’s reformulation of its drug candidate DT-216 for Friedrich ataxia will delay a potential commercial ...

PLANO, Texas--(BUSINESS WIRE)-- Reata Pharmaceuticals, Inc. (Nasdaq: RETA), (“Reata,” the “Company,” “our,” “us,” or “we”), a clinical-stage ...

Reata Pharmaceuticals has done its homework in responding to the FDA’s concerns about an application for its nerve disorder drug. But now the biotech has to sit tight for an extra three months while ...

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 ...

A team of researchers has developed a new method to screen FDA-approved drugs to determine if they could be repurposed or improved to help patients with a rare, debilitating disease of the nervous ...

(BHVN) closed 7% higher on Wednesday on the heels of hitting enrollment milestones in two high-priority clinical programs, ...