Megan Garlapow, PhD

All articles by Megan Garlapow, PhD

• New steroid alternative helps boys with Duchenne maintain growth

  The steroid alternative vamorolone appeared safe and effective in boys with Duchenne muscular dystrophy (DMD) aged 7 to 17.

• Gene therapy GNT0004 shows lasting benefit for boys with Duchenne

  A one-time gene therapy was linked to stable or improved movement in boys with Duchenne muscular dystrophy.

• PTC Therapeutics withdraws ataluren application after FDA review

  The FDA determined that current data for ataluren in Duchenne muscular dystrophy (DMD) are unlikely to meet approval standards.

• Genetic clues emerge for heart damage in Duchenne muscular dystrophy

  Certain genes may help explain why heart disease is worse in some people with Duchenne muscular dystrophy.

• Two-year study shows lasting gains from Elevidys gene therapy for DMD

  A single gene therapy dose helped young boys with Duchenne muscular dystrophy keep their strength longer than expected.

• 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).