The American Journal of Managed Care13d
Later Loss of Ambulation Linked With Better Heart Health in DMD
The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with ...
precisionmedicineonline2d
Belief BioMed's Duchenne Muscular Dystrophy Gene Therapy Gets FDA IND Clearance
The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.
The American Journal of Managed Care12d
Quantifying the Altruism Value for a Rare Pediatric Disease: Duchenne Muscular Dystrophy
Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...
The Miami Times13d
First female in the world receives revolutionary new gene therapy for muscular dystrophy in Miami
When Raniya Scott was just two years old, her world changed with an unexpected diagnosis: Duchenne muscular dystrophy (DMD), ...
Business Insider10d
Santhera Receives Positive NICE Final Guidance for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy
The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study ...