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Insmed’s ASCEND Study and INS1201, an Investigational Gene Therapy for Duchenne Muscular Dystrophy
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Solid plans to seek FDA guidance in 2026 for Duchenne Gene Therapy Candidate SGT-003 S
Solid Biosciences today reported an update on the progress of the INSPIRE DUCHENNE open label, Phase I/II clinical trial with SGT-003 and their planned regulatory discussions in 2026. 23 Pediatric […]
Letter from Italfarmaco about patient deaths while taking givinostat
Italfarmaco issues a letter to address the recent online discussions about the individuals who have passed away while being treated with Duvyzat (givinostat), confirming that they have determined the deaths […]
ITF Therapeutics shares publication of long-term data showing givinostat can delay loss of functional abilities in Duchenne
Results published in the Annals of Clinical and Translational Neurology show that long-term treatment with givinostat, a histone deacetylase (HDAC) inhibitor, in combination with corticosteroids, delayed disease progression in ambulant individuals with […]
ELEVIDYS UPDATE
Breaking News: Sarepta to resume shipments of ELEVIDYS for ambulatory individuals with Duchenne 07/28/25 5:09 PM EDT We are pleased to report that the US FDA has recommended that Sarepta […]
Important Update: Sarepta Therapeutics Announces Strategic Restructuring and Pipeline Prioritization Plan to Maintain Long-term, Sustainable Growth and Provides Update on ELEVIDYS Label
CureDuchenne is sharing a community letter from Sarepta announcing a strategic company restructuring and pipeline prioritization plan. Additionally, Sarepta shared that the U.S. FDA has requested and Sarepta has agreed to include a […]
Edgewise Therapeutics Reports Positive Results on Sevasemten Program
Edgewise Therapeutics, which received early funding from CureDuchenne, has reported positive results in its sevasemten program for Becker and Duchenne muscular dystrophies. New open-label data in Becker from the MESA trial demonstrated […]
Regulatory Update on Capricor’s Deramiocel for Duchenne
Capricor’s Deramiocel for Duchenne: Capricor Therapeutics, which received early funding from CureDuchenne, has indicated that the FDA has decided that an Advisory Committee meeting is not warranted at this time. […]
CureDuchenne’s latest investment aims to address the limitations of current AAV-delivered gene therapy treatments for Duchenne muscular dystrophy (DMD).
Our recent investment in Entos Pharmaceuticals (https://entospharma.com) aims to explore the potential of their non-viral, redosable Fusogenix PLV platform to deliver full-length dystrophin protein to all muscle groups in a […]