There is a simple discount patient access scheme for vamorolone. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact ...
Muscular dystrophy can appear in infancy up to middle age or later, and its form and severity are determined in part by the age at which it occurs. Some types of muscular dystrophy typically ...
The FDA has granted a Rare Pediatric Disease Designation (RPDD) to HORA-PDE6b (eyeDNA Therapeutics), a novel gene therapy, for the treatment of patients with inherited retinal dystrophy (IRD) caused ...
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