Megan Garlapow, PhD

All articles by Megan Garlapow, PhD

• Researchers examine how to improve safety of gene therapy for Duchenne

  Gene therapy for Duchenne muscular dystrophy (DMD) shows real promise, but high doses have caused rare serious side effects.

• HHS adds Duchenne to newborn screening panel

  Adding DMD and metachromatic leukodystrophy to newborn screening can help identify affected babies before symptoms appear.

• DMD RNA imbalance may limit benefits of genetic therapy

  Uneven dystrophin RNA production is common in DMD and may limit how much dystrophin current genetic therapies can restore in muscle.

• New data indicates DYNE-251 boosts dystrophin and preserves function

  A new treatment candidate for DMD increased dystrophin levels and improved movement while keeping lung function steady.

• Glucocorticoids found to improve thinking ability in a Duchenne mouse model

  A steroid improved memory and brain cell connections in a mouse model of Duchenne muscular dystrophy (DMD).

• 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.