Megan Garlapow, PhD

All articles by Megan Garlapow, PhD

• Ruxolitinib extends lifespan and strengthens muscle in Duchenne model

  Ruxolitinib reduced harmful senescent cells and improved muscle and bone in a mouse model of Duchenne muscular dystrophy.

• Microdystrophin gene therapy improves muscle function but raises heart risks

  A gene therapy called GNT0004 improved muscle and heart function in DMD but caused heart rhythm problems at high doses.

• New stem cell therapy to be tested in patients with Duchenne muscular dystrophy

  A new cell therapy called MyoPAXon has entered early testing in patients with Duchenne muscular dystrophy.

• New research demonstrates lasting benefits of tRNA therapy in DMD models

  Early research indicates the therapy could restore nearly 70% of healthy dystrophin protein in people with DMD caused by nonsense mutations.

• 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.