Personalized therapy for rare genetic diseases: Patient-derived organoids offer new hope
Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...
precisionmedicineonline1d
Belief BioMed's Duchenne Muscular Dystrophy Gene Therapy Gets FDA IND Clearance
The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.
The Pharma Letter1d
RPD designation for Nippon Shinyaku’s DMD candidate
The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to NS-051/NCNP-04, which is ...
Gene therapies have been uneven for DMD — but these companies hope to turn the tide
Despite mixed results using gene therapies to treat Duchenne muscular dystrophy, drug developers are pushing ahead with the ...
Muscular Dystrophy News2d
FDA awards fast track designation to DM1 treatment DYNE-101
The FDA has granted fast track designation to DYNE-101, a therapy for myotonic dystrophy type 1 (DM1) now in testing in a ...
bangaloremirror.indiatimes2d
Toddler’s battle with DMD: Hope rests on gene therapy
According to Dr Ann Agnes Mathew, Consultant Pediatric Neurologist at Bangalore Baptist Hospital, DMD is life-threatening and ...
themarijuanaherald3d
Full-Spectrum CBD Oil Reduces Key Markers of Muscle Damage in Duchenne Muscular Dystrophy
A new study published in the peer-reviewed journal Muscle & Nerve shows that full-spectrum cannabidiol (CBD) oil may help ...
Sentinel-Tribune3d
Perrysburg gymnasts fundraise for Duchenne Muscular Dystrophy
The Perrysburg gymnastics team hosted the area’s only All Around Invitational on January 20 — the Noah Ninja’s meet: ...
Solid Biosciences: Differentiating Factors Of SGT-212 To Set Itself Apart
Initiation of Phase 1b study using SGT-212 for the treatment of patients with Friedreich's Ataxia expected in 2H25. Click ...
Muscular Dystrophy News4d
Corticosteroid at low dose may help with muscle damage, inflammation
A pilot study in 19 LGMD patients and one with Becker MD found low-dose prednisone helped lower markers of muscle damage and ...
Dyne Therapeutics Receives FDA Fast Track Designation for DYNE-101 for the Treatment of Myotonic Dystrophy Type 1
Company Anticipates Submitting for U.S. Accelerated Approval in H1 2026 -WALTHAM, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, ...
Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia
Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track ...