The gene is located on the X chromosome ... This provides a starting point for a therapy," says Falcucci. Didier Stainier, ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

The Muscular Dystrophy Association celebrates the FDA approval of Evrysdi in tablet form, a more accessible treatment option ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

The permanent appointment of the Perelman School of Medicine dean is subject to the approval of the Penn board of trustees.

Astellas has invested heavily in novel technologies that are not necessarily the most successful or popular in the industry.

Boehringer will leverage ExpressionEdits' Genetic Syntax Engine which generates optimized introns to increase protein expression.

ExpressionEdits' Genetic Syntax Engine platform is designed to enhance gene expression within gene therapies by restoring introns.

Orchard Therapeutics has tasked Robin Kenselaar with accelerating growth of Lenmeldy, the world’s most expensive drug, ...

GenSight Biologics Announces Five-Year Efficacy and Safety Results for LUMEVOQ® Gene Therapy at the Conclusion of the REFLECT Study ...

Kyle Registre's childhood dream of becoming a pilot can finally come true now that he says he's been cured of sickle cell complications.