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Solid Biosciences Plans to Request a Meeting with the FDA in mid-2025 to Discuss Potential Accelerated Approval Pathways for SGT-003

GenAssist Announces FDA Approval of New Drug Application for GEN6050X Exon 50 Skipping Therapy

Capricor Therapeutics Announces FDA Acceptance and Priority Review of its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy

FDA Grants Rare Pediatric Disease Designation to Nippon Shinyaku’s Exon 51 Skipping Study (NS-051/NCNP-04)

3 Drugs Receiving Accelerated Approval from the FDA and Reviewed by the Office of Inspector General Raised Concerns

The FDA Designates IPS HEART’s GIVI-MPC Stem Cell Therapy for Becker Muscular Dystrophy an Orphan Drug

What is Ataluren (Translarna)? What is Ataluren Used For?

Approved Therapies

What is Agamree (vamorolone)? What is Agamree Used For?

Approved Therapies

Frequently Asked Questions About Eteplirsen (Exondys 51) for DMD Treatment

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Frequently Asked Questions About Elevidys Used for DMD

Can Ekinci - August 21, 2026

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Del-Zota SAFARI44 Phase 3 Trial: A New Exon 44 Skipping Study for Duchenne Muscular Dystrophy

RGX-202 Duchenne Gene Therapy Shows NSAA Improvement and Microdystrophin Expression — But Important Biomarker Data Missing

CRISPR GuideX Tool: hfCas12Max in CRISPR-Based Therapeutics

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