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On May 15, Pfizer announced positive results from a phase 1b trial assessing treatment with the company’s investigational gene therapy PF-06939926 in ambulatory boys with Duchenne muscular dystrophy (DMD). Preliminary…

On May 22, Danish company Orphazyme announced Fast Track designation from the US Food and Drug Administration (FDA) for development of the investigational drug arimoclomol for the treatment of amyotrophic…

MDA Let’s Play takes family fun online

A Q&A with Priya Sunil Kishnani, MD, MBBS

Highlights from the virtual meeting with researchers and clinicians

Experts weigh in on the keys to resilience in a stressful world

The Zelaya family (clockwise from left): Bevsi (amazing mom), Jaime (advocate dad), Josiah (kind big brother), Nehemiah (curious younger brother), and Leah (compassionate middle sister) Why we advocate for families…

How to master your tasks and practice self-care

MDA Care Center physicians across the country are working on the frontlines of the novel coronavirus pandemic. Every day, they’re fielding patient questions and finding new ways to practice medicine…

Over the course of the last several months, the US Food and Drug Administration (FDA) has undertaken many actions aimed at slowing the spread of the novel coronavirus and ensuring…