Rare and Genetic Disease Network


Sarepta Presents Positive Gene Therapy Data for Duchenne Muscular Dystrophy

Sarepta Therapeutics announced that at the 23rd International Congress of the World Muscle Society in Mendoza, Argentina, Jerry Mendell, MD, of Nationwide Children’s Hospital presented positive updated results from its gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne muscular dystrophy (DMD).

FDA Approves Antibacterial Drug for Rare Lung Disease

The U.S. Food and Drug Administration approved a new drug, Arikayce (amikacin liposome inhalation suspension), for the treatment of lung disease caused by a group of bacteria, Mycobacterium avium complex (MAC) in a limited population of patients with the disease who do not respond to conventional treatment (refractory disease).

Rare Disease Advocacy

Jodie Gillon Global Medical Lead, Patient Engagement Rare Diseases at Pfizer discusses Pfizer’s role in patient advocacy.

Vascular Ehlers-Danlos Syndrome (vEDS)

William T. Andrews, MD, FACP, Chief Medical Officer of Acer Therapeutics discusses his company’s focus on Vascular Ehlers-Danlos Syndrome (vEDS), an inherited connective tissue disorder that is caused by defects in a protein called collagen.

Special Reports