The American Journal of Managed Care1d
SGT-003 Gene Therapy Shows Potential in DMD
In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...
BioSpace5h
6 Rare Diseases That Have New FDA-Approved Treatments
ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...
FierceBiotech3d
Entrada's 2-year ordeal ends as FDA lifts hold on DMD drug
Entrada Therapeutics is finally able to get its Duchenne muscular dystrophy candidate (DMD) back on track after the FDA ...
The American Journal of Managed Care13d
Adults With DMD More Than Twice as Likely to Experience Fractures
Less than half of patients had undergone bone monitoring, suggesting a need for better clinical guidance and management of ...
Children denied access to life-changing drug due to paperwork, say families
Givinostat should be available to people with Duchenne muscular dystrophy through a type of compassionate use scheme.
DMD market expected to grow to $5.2bn in the 7MM by 2033
The Duchenne muscular dystrophy (DMD) landscape across the seven major markets (7MM) - the US, France, Germany, Italy, Spain, ...
BBC on MSN9d
'It's cruel that I cannot get muscular dystrophy drug'
A 12-year-old boy with a severe form of muscular dystrophy has said it is "cruel" that a new drug is not being made available ...
FierceBiotech9d
Solid Bio's stock soars as early DMD gene therapy data beat expectations
Solid Biosciences has surged back into the Duchenne muscular dystrophy (DMD) gene therapy race. | Solid Biosciences has ...
Precision BioSciences Announces Oral Presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
Hosted on MSN8d
What To Know About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...