Riya Ajmera

All articles by Riya Ajmera

• DT-DEC01 shows promise as a treatment for all patients with DMD

  All three patients experienced improvements in various aspects of muscle function, heart and lung health, and daily movement.

• Late-stage study suggests DMD gene therapy improves muscle integrity in boys

  Delandistrogene moxeparvovec is designed to counter muscle breakdown by delivering dystrophin to muscle cells.

• New gene therapy shows promise in slowing or stabilizing DMD in young boys

  Better mobility and sustained functional dystrophin levels were seen in young boys with DMD treated with delandistrogene moxeparvovec.

• Transitioning from pediatric to adult care with a neuromuscular disease

  Without a structured transition plan, young adults with neuromuscular diseases may face significant risks.

• The old, the new, and the uncertainty in genetic testing

  Emerging genetic testing technologies include optical genome mapping, long-read sequencing, and RNA sequencing.

• Expert: Neuromuscular diseases are not ‘one size fits all’

  “Even with the same disease state, the phenotype is very different,” and should influence treatment strategies, an expert said.

• Newborn screening for DMD and Pompe expected to become more widespread

  Only 4 states have approved newborn screening for Duchenne muscular dystrophy.

• MDA working to improve air travel for wheelchair users

  The federal Department of Transportation in 2024 finalized a rule that enhances air travel experiences for wheelchair users.