Megan Garlapow, PhD

All articles by Megan Garlapow, PhD

• Study finds gene therapy heart risks are rare and manageable

  Heart inflammation linked to gene therapy for Duchenne muscular dystrophy (DMD) is uncommon and usually mild and reversible.

• New review highlights progress and cautions in AAV gene therapy for DMD

  Gene therapy for Duchenne muscular dystrophy (DMD) is advancing quickly, but important safety risks still need to be carefully managed.

• 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.