14.10.2022 04:47:46
|
Reata : FDA Doesn't Plan To Hold Meeting To Discuss NDA For Omaveloxolone For Friedreich's Ataxia
(RTTNews) - The U.S. Food and Drug Administration has informed Reata Pharmaceuticals Inc. (RETA) that it does not plan to hold an advisory committee meeting in connection with its review of the company's New Drug Application for omaveloxolone for the treatment of patients with Friedreich's ataxia, the company said in a statement.
Friedreich's ataxia is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder typically caused by a trinucleotide repeat expansion in the first intron of the frataxin gene, which encodes the mitochondrial protein frataxin.
Pathogenic repeat expansions can lead to impaired transcription and reduced frataxin expression, which can result in mitochondrial iron overload and poor cellular iron regulation, increased sensitivity to oxidative stress, and impaired mitochondrial ATP production.
Patients with Friedreich's ataxia typically experience symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue that commonly results in motor incapacitation with patients requiring a wheelchair in their 20s.
Wenn Sie mehr über das Thema Aktien erfahren wollen, finden Sie in unserem Ratgeber viele interessante Artikel dazu!
Jetzt informieren!
Nachrichten zu Reata Pharmaceuticals Inc (A)mehr Nachrichten
Keine Nachrichten verfügbar. |