Megan Garlapow, PhD

All articles by Megan Garlapow, PhD

• Sarepta advances study aimed at reducing liver injury risk in gene therapy

  The FDA authorized Sarepta to test whether using sirolimus before gene therapy can lower the risk of acute liver injury in nonambulatory DMD.

• Targeted gene therapy boosts breathing strength in Duchenne model

  Targeting breathing muscles improved dystrophin levels and reduced damage with fewer liver risks in a mouse model of DMD.

• FDA approves updated Elevidys safety and prescribing information

  Patients receiving delandistrogene moxeparvovec (Elevidys) will face stricter monitoring and new safety warnings.

• Research on key gene networks expands understanding of DMD

  Research showed five major genes driving inflammation, immune imbalance and scarring in Duchenne muscular dystrophy.

• 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.