In honor of Charcot-Marie-Tooth (CMT) Awareness Month, the Muscular Dystrophy Association joins the Charcot-Marie-Tooth Association (CMTA) and the Hereditary Neuropathy Foundation (HNF) to host an externally led patient-focused drug development (PFDD) meeting for CMT.
The PFDD meeting is being held on Friday, Sept. 28 at the College Park Marriott Hotel and Conference Center in Hyattsville, Md.
The meeting will bring together patients, caregivers, industry, healthcare providers and National Institutes of Health and U.S. Food and Drug Administration representatives to understand the challenges of patients living with CMT and the unmet medical need in a disease where there are no drug treatments are currently available.
A live webcast will begin at 8:30 a.m. ET and will be available at https://www.hnf-cure.org/pfddlive/.
Additionally, the HNF, CMTA and MDA will be collaborating on a “Voice of the Patient” report for the FDA that summarizes the input provided by patients and patient representatives at this meeting. This partnership will ensure that therapies and treatments address the concerns of the CMT community. Under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), these meetings are designed to more systematically gather patients’ perspectives on their condition and available therapies to treat their condition.
We thank HNF for coordinating this important meeting and the CMTA for their partnership. We believe this collaboration will help accelerate research and treatments for CMT patients worldwide.