News about "Duchenne muscular dystrophy "

Atossa Therapeutics Receives FDA Orphan Drug Designation for Duchenne Therapy

Atossa Therapeutics Receives FDA Orphan Drug Designation for Duchenne Therapy

Atossa Therapeutics has received US Food and Drug Administration (FDA) Orphan Drug Designation (ODD) for (Z)-Endoxifen for the treatment of Duchenne Muscular Dystrophy, further supporting the company’s development programme in rare paediatric neuromuscular diseases.

Duchenne Muscular Dystrophy | 17/01/2026 | By News Bureau

Sarepta Pauses Shipments of Duchenne Gene Therapy Elevidys Amid FDA Safety Review

Sarepta Pauses Shipments of Duchenne Gene Therapy Elevidys Amid FDA Safety Review

Sarepta Therapeutics has announced a voluntary and temporary suspension of all US shipments of its Duchenne muscular dystrophy (DMD) gene therapy, ELEVIDYS (delandistrogene moxeparvovec), following the FDA’s distribution suspension request last week.

Duchenne Muscular Dystrophy | 24/07/2025 | By Dineshwori 375

Somite Therapeutics Receives FDA Orphan Drug and RPDD Designations for SMT-M01

Somite Therapeutics Receives FDA Orphan Drug and RPDD Designations for SMT-M01

Somite Therapeutics' SMT-M01 program leverages the company's proprietary AlphaStem AI platform to develop a novel cell replacement therapy for DMD.

Duchenne Muscular Dystrophy | 17/09/2024 | By Aishwarya 664


 

 

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