RDR Investigator-Sponsored Trial Treats First Patient

It is with great pleasure and enthusiasm that we at Rare Disease Research announce the enrollment of the first patient in our Investigator-Sponsored Trial for boys with Duchenne muscular dystrophy. The phase 4 trial entitled “An Open-Label Study to Evaluate the Safety of Golodirsen in Non-Ambulant Patients with Duchenne Muscular Dystrophy” seeks to enroll 12 non-ambulant subjects, 7 years of age or older, with a confirmed genetic mutation amenable to treatment by exon 53 skipping.

Patients will receive weekly infusions of golodirsen, an FDA-approved therapy for ambulant subjects with mutations amenable to exon 53 skipping.  

We are excited to bring this study to all eligible patients. It is our goal to facilitate access to investigational treatments while aiding in cutting-edge research.

Contact us

For more information, contact us at: 
Rare Disease Research, LLC 
Study Coordinator: Sarah Mackett 
678 - 883 - 6897
sarah.mackett@rarediseaseresearch.com