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After Elevidys: DMD Advocacy Past First Gene Therapy Approval

For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.

Sharon hesterleeMichael kellyJeffrey chamberlainUniversity of washington school medicineWorld muscle societyMuscular dystrophy associationInternational congressSarepta therapeuticNorth star ambulatory assessmentParent project muscular dystrophyMuscular dystrophy specialized research centerWashington schoolCapricor therapeuticFirst gene therapyTreat duchenne muscularAnnual international congress

Muscular Dystrophy Association Announces Collaboration with

New York, Oct. 30, 2023 (GLOBE NEWSWIRE) Muscular Dystrophy Association (MDA) and Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced their.

United statesNew yorkTanabe pharma americaLou gehrigYasutoshi kawakamiSharon hesterleeMitsubishi tanabe pharma corporationMitsubishi tanabe pharma america incMuscular dystrophy associationMitsubishi chemical groupCare centersDystrophy associationMitsubishi tanabe pharma americaMuscular observationalClinical outcomes assessmentChief research officer

Novel Corticosteroid Approved for Rare Disease

Vamorolone gets a nod from FDA for Duchenne muscular dystrophy

Vamorolone agamreeSharon hesterleeJudy georgeCatalyst pharmaceuticalsMuscular dystrophy associationMedpage today

FDA Okays Drug for Duchenne Muscular Dystrophy

Vamorolone (Agamree) is a structurally unique steroidal anti-inflammatory drug with fewer side effects than traditional corticosteroids.

Sharon hesterleeDrug administrationCatalyst pharmaceuticalsMuscular dystrophy associationMuscular dystrophyUchenne muscular dystrophy dmdAdverse effectsSide effectsPatient safetyBone densityUs food and drug administrationNited states food and drug administrationOrphan drugsShort statureWeight gainWeight management

Muscular Dystrophy Association-Supported Drug AGAMREE®

New York, Oct. 26, 2023 (GLOBE NEWSWIRE) The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of.

North carolinaUnited statesNew yorkDuke universityEjay griffinEric hoffmanBarry byrneReveragen biopharmaEdward smithLouis kunkelSharon hesterleeCatalyst pharmaceuticalsCare center at duke universityResearch developmentMuscular dystrophy associationResource center

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