welcome to DuchenneXchange

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

Fireside Chat at the 13th Orphan Drugs and Rare Diseases Global Congress – Americas 2020


A lively discussion began with Dr. Marks expressing that one of the challenges COVID has presented to him was that progress on building new frameworks to treat rare diseases was interrupted. He expects that they’ll be able to get back on track now that progress has been made on COVID-19 vaccines and treatments, although it may be “a bumpy ride over the next few months.”

Speaking about Duchenne on the behalf of the rare disease community, Debra noted that “when you have an abbreviated life span, taking a year out of a person’s life is tragic” and that people with rare diseases have been and continue to be in a state of emergency, drawing parallels between the nation’s ability to respond to COVID and what ought to be done for rare diseases.