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FDA
Press Releases
GenAssist Announces FDA Approval of New Drug Application for GEN6050X Exon 50 Skipping Therapy
Press Releases
Capricor Therapeutics Announces FDA Acceptance and Priority Review of its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy
Editorials
Clinical Trials Review of Elevidys Gene Therapy: Is Elevidys Cost Worthy?
Approved Treatments
FDA-Approved Treatments for DMD (Duchenne Muscular Dystrophy)
Press Releases
FDA Grants Rare Pediatric Disease Designation to Nippon Shinyaku’s Exon 51 Skipping Study (NS-051/NCNP-04)
Press Releases
3 Drugs Receiving Accelerated Approval from the FDA and Reviewed by the Office of Inspector General Raised Concerns
Research
The FDA Designates IPS HEART’s GIVI-MPC Stem Cell Therapy for Becker Muscular Dystrophy an Orphan Drug
Research
What is Ataluren (Translarna)? What is Ataluren Used For?
Approved Treatments
What is Agamree (vamorolone)? What is Agamree Used For?
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Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy
DMD Warrior
-
December 26, 2024
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Editorials
Accredited Duchenne Centers (ADC) Aims to Provide Uniform and Up-to-date Care for All People Living with Duchenne Muscular Dystrophy
Research
RGX-202 Gene Therapy Phase 3 Clinical Trial Begins
Research
Entrada Therapeutics Begins Clinical Trials in Europe for Exon 44 and Exon 45 Skipping Therapies