Tag: Muscular Dystrophy
FDA Expands Approval for Duchenne Muscular Dystrophy Gene Therapy
Approval allows for use in nonambulatory individuals ≥4 years with a confirmed gene mutation
FDA Approves Generic Emflaza Oral Suspension for Duchenne Muscular Dystrophy
Approval of deflazacort oral suspension is for patients 5 years of age and older
FDA Approves First Gene Therapy to Treat Duchenne Muscular Dystrophy
The recombinant gene therapy is administered in a single intravenous dose
Vamorolone Effective, Safe for Boys With Duchenne Muscular Dystrophy
Bone morbidities reduced, efficacy retained with vamorolone for boys with Duchenne muscular dystrophy
Home Monitoring of Lung Function Feasible in Teens With Duchenne
Peak flow meter handheld device useful for adolescents with Duchenne muscular dystrophy
FDA Approves Emflaza for Duchenne Muscular Dystrophy
Approved to treat Duchenne muscular dystrophy in patients 5 years and older
FDA Approves First Drug for Duchenne Muscular Dystrophy
Exondys 51 sanctioned for patients with mutation of the dystrophin gene amenable to exon 51 skipping
FDA Panel Votes Against Approval of Eteplirsen for DMD
Clinical data did not meet the agency's requirements for well controlled studies necessary for approval
IV Bisphosphonate Tx Linked to Drop in Bone Turnover in DMD
Need for therapies with less bone turnover suppression in Duchenne muscular dystrophy
