welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!

Pfizer Says No New Serious Adverse Events in DMD Gene Therapy Trial

Drugmaker Pfizer Inc said on Tuesday that it had administered doses of its experimental gene therapy for rare degenerative muscular condition Duchenne muscular dystrophy (DMD) to an additional 9 boys, with no serious adverse events observed.

The company plans to start a pivotal trial on the gene therapy in ambulatory patients in the next several weeks, Seng Cheng, chief scientific officer of Pfizer’s rare disease unit said during a presentation to investors. The company modified the regimen for the drug earlier this year after three patients had to be hospitalized.


Pfizer Says No New Serious Adverse Events In DMD Gene Therapy Trial