Megan Garlapow, PhD

All articles by Megan Garlapow, PhD

• 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.

• FDA clears way for Sarepta to restart Elevidys shipments

  Patients with DMD who can still walk will regain access to Elevidys as Sarepta resumes shipments following FDA clearance.

• Micro-dystrophin gene therapy improves survival but may pose long-term risks

  Micro-dystrophin gene therapy greatly extended lifespan and improved heart function in severe DMD preclinical models.

• PBGENE-DMD receives Rare Pediatric Disease Designation from FDA

  PBGENE-DMD could help up to 60% of patients with Duchenne muscular dystrophy by permanently editing their dystrophin gene.

• New gene therapy delivery approach restores full-length dystrophin in DMD mice

  A new gene therapy using triple AAV vectors restored full-length dystrophin and improved muscle function in mice with DMD.

• Prenatal stress shapes future health in mouse model of DMD

  Stress during late pregnancy changed how Duchenne muscular dystrophy (DMD) impacted offspring in a mouse model.

• Gene therapy Elevidys expands hope for patients with Duchenne

  Elevidys is a one-time gene therapy that helps patients with DMD produce a key muscle protein and is under ongoing safety evaluation.