Solid Biosciences Announces Duchenne Muscular Dystrophy Added to National Recommended Uniform Screening Panel by the U.S. Department of Health and Human Services (Bio Space)
Solid Biosciences Inc., which acquired UF startup AavantiBio, shared that the U.S. Department of Health and Human Services (HHS) officially added Duchenne muscular dystrophy (Duchenne) to the Recommended Uniform Screening Panel (RUSP), the list of conditions recommended for universal newborn screening across the United States.
Solid Biosciences Awarded Innovation Passport Designation Under the New UK Innovative Licensing and Access Pathway for SGT-003 (BioSpace)
Solid Biosciences Inc., which acquired UF startup AavantiBio, announced that SGT-003, the Company’s investigational gene therapy for Duchenne muscular dystrophy (Duchenne), has been granted an Innovation Passport under the new ILAP.
Does My Child Have Duchenne Muscular Dystrophy? Take the Quiz.
Duchenne muscular dystrophy — sometimes referred to simply as Duchenne — is a genetic disorder that causes progressive muscle loss and weakness. It occurs as a result of gene mutations involved in the production of a protein called dystrophin, according to the Muscular Dystrophy Association (MDA).
Dr. Barry Byrne Presents Improvements in Key Duchenne Muscular Dystrophy Measures, Microdystrophin Expression
Data presented by Barry Byrne, MD, Ph.D. suggest that intravenous SGT-001 (Solid Biosciences) is well-tolerated and efficacious in DMD.
Parent Project Muscular Dystrophy Awards $348,891 Grant to University of Florida Wellstone Center
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy, awarded the University of Florida Wellstone Muscular Dystrophy Cooperative Center a grant for $348,891.
New $6.25 Million Grant Allows UF To Expand 10-Year Study of Muscular Dystrophy Disease Progression
A new five-year, $6.25 million grant from the National Institutes of Health will support the University of Florida’s continued efforts to develop MRI biomarkers for individuals with Duchenne muscular dystrophy, and expand the study to include people with Becker muscular dystrophy.
University of Florida Received £500,000 Grant From Duchenne UK to Overcome Immunity Challenges in Gene Therapy
Duchenne UK has partnered with one of the world’s leading gene therapy teams focusing on AAV immunology. Drs. Manuela Corti and Barry Byrne at the Powell Gene Therapy Center, University of Florida, will try to temporarily modify the immune system so that anybody can be treated with gene therapy, and, if necessary, can receive repeat […]
What Causes Duchenne Muscular Dystrophy? UF Researcher Explains
Duchenne muscular dystrophy, a genetic disease characterized by progressive muscle weakness, is present at birth in people who have the condition. In people with Duchenne, the muscles lack a protein called dystrophin, which is critical for muscle function and repair. While all people with Duchenne share a lack of or defective dystrophin, the condition can […]