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Duchenne Muscular Dystrophy News
FDA Expands Approval for Duchenne Muscular Dystrophy Gene Therapy
MONDAY, June 24, 2024 – The U.S. Food and Drug Administration has expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular...
Sarepta Therapeutics Announces Expanded US FDA Approval of Elevidys to Duchenne Muscular Dystrophy Patients Ages 4 and Above
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 20, 2024-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug...
FDA Approves Oral Duvyzat for Duchenne Muscular Dystrophy
MONDAY, March 25, 2024 – The U.S. Food and Drug Administration has approved Duvyzat (givinostat) as an oral medication for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of...
FDA Approves Duvyzat (givinostat) for Duchenne Muscular Dystrophy
MILAN--(BUSINESS WIRE)-- Mar 22, 2024 – Italfarmaco S.p.A. announced today that the U.S. Food and Drug Administration (FDA) has approved Duvyzat™ (givinostat), a novel histone deacetylase (HDAC) i...
In Early Research, Heart Drugs Show Promise in Fighting Muscular Dystrophy
WEDNESDAY, Jan. 3, 2024 – A regularly prescribed class of heart medications might be capable of treating one of the most common forms of muscular dystrophy, a new study in mice suggests. Myotonic...
Monthly News Roundup - October 2023
FDA Clears Loqtorzi as First Treatment for Nasopharyngeal Carcinoma (NPC) In October, the U.S. Food and Drug Administration (FDA) approved Coherus BioSciences’ Loqtorzi (toripalimab-tpzi) to treat a...
FDA Approves Agamree (vamorolone) for the Treatment of Duchenne Muscular Dystrophy
CORAL GABLES, Fla., Oct. 26, 2023 (GLOBE NEWSWIRE) – Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX) today reported that Santhera Pharmaceuticals ("Santhera") has obtained...
FDA Approves Elevidys (delandistrogene moxeparvovec-rokl) Gene Therapy to Treat Duchenne Muscular Dystrophy
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 22, 2023-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug...
FDA Approves Amondys 45 (casimersen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 45
CAMBRIDGE, Mass., Feb. 25, 2021 (GLOBE NEWSWIRE) – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug ...
FDA Approves Viltepso (viltolarsen) for the Treatment of Duchenne Muscular Dystrophy in Patients Amenable to Exon 53 Skipping Therapy
PARAMUS, N.J., Aug. 12, 2020 /PRNewswire/ – NS Pharma, Inc. announced today that the U.S. Food & Drug Administration (FDA) has approved Viltepso (viltolarsen) injection for patients with Duchenne...
FDA Approves Vyondys 53 (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53
CAMBRIDGE, Mass., Dec. 12, 2019 (GLOBE NEWSWIRE) – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug...
PTC Therapeutics Receives FDA Approval for the Expansion of the Emflaza (deflazacort) Labeling to Include Patients 2-5 Years of Age
SOUTH PLAINFIELD, N.J., June 7, 2019 /PRNewswire/ – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) approved the company's supplemental New...
FDA Approves Emflaza (deflazacort) to treat Duchenne Muscular Dystrophy
February 9, 2017 – The U.S. Food and Drug Administration today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy...
FDA Approves Exondys 51 (eteplirsen) for Duchenne Muscular Dystrophy
September 19, 2016 – The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys...
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