Megan Garlapow, PhD

All articles by Megan Garlapow, PhD

• NS-051/NCNP-04 granted FDA Orphan Drug designation for DMD

  Patients with Duchenne muscular dystrophy may benefit from a potential therapy now supported by an FDA orphan designation.

• Fractures and growth delays remain common in boys with Duchenne

  Half of boys with Duchenne muscular dystrophy had vertebral fractures, many of which happened silently without obvious trauma.

• New data suggests del-zota reverses muscle decline in Duchenne

  In a one-year trial, del-zota helped boys with DMD gain strength and function, reversing their expected decline.

• Study finds age and steroid use drive bone loss in Duchenne muscular dystrophy

  Children with DMD experience steady bone loss as they age and continue steroid treatment, raising concerns for long-term mobility.

• Experts set standards to guide safer Duchenne gene therapy

  The Muscular Dystrophy Association and Parent Project Muscular Dystrophy released guidelines to help safely use gene therapy for DMD.

• Patients face new challenges as Duchenne gene therapies advance

  Recent gene therapies for Duchenne muscular dystrophy offer hope but also bring risks, including patient deaths.

• Newborn screening for Duchenne muscular dystrophy offers earlier answers

  Earlier detection of DMD through newborn screening could give families more time to access treatment and plan care.

• New review highlights the complexity of treating cognitive aspects of DMD

  One-third of individuals with DMD experience cognitive or behavioral differences, but treating DMD’s impact on the brain poses challenges.

• Review explores delivery of DMD therapies to the central nervous system

  New RNA-based treatments show promise for improving brain symptoms in DMD by reaching the central nervous system. 

• Researchers expand hope for DMD while grappling with setbacks

  Advances in gene and small molecule therapies offer new hope for Duchenne muscular dystrophy, but no cure currently exists.