The State of Illinois is recognizing our 20th Annual Connect Conference in Chicago, Illinois, June 26-29, 2014 by declaring the week of June 23 as Duchenne Muscular Dystrophy Awareness Week! With over 500 families and Duchenne experts expected…

PPMD will convene a pre-conference meeting of physical therapists, occupational therapists, and clinical coordinators who specialize in the research, assessment, longitudinal data collection, and treatment of people with Duchenne, June 25 and 26 in Chicago. This meeting will precede…

Children's Hospital Colorado (Children's Colorado) was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne).

Duchenne is the most…

The Goldwater Institute of Arizona has designed state model legislation, known as a "Right to Try" bill, which would make experimental drugs available to terminally-ill patients without the FDA's approval. Under this legislation, a patient with "an advanced stage of a disease with an unfavorable prognosis and no known cure" could obtain an experimental drug that has passed…

Phase 1b/2a Clinical Program Resumes Enrollment and Dosing in DMD Patients 

Preliminary Clinical Data to be presented at Upcoming Medical Conference 

Read the news release from DART Therapeutics:

DART Therapeutics Continues Clinical Development of HT-100 for Duchenne Muscular Dystrophy 

Cambridge, Mass.—June 12,…

Prosensa Plans to Submit a New Drug Application to the FDA this Year; Dialogue with EMA continues with intent to seek approval

More good news from the FDA! This news from Prosensa continues to demonstrate that FDA recognizes the urgent needs of this community, the importance of the patient voice to understand the benefit/risk assessment, and continues to…