We’re thrilled to share a major breakthrough for the Charcot-Marie-Tooth (CMT) community! NMD Pharma’s investigational drug, NMD670, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). This designation is awarded to therapies with the potential to address rare diseases, like CMT, and comes with benefits that can help accelerate its development.
Why This Matters:
Currently, there are no approved treatments for CMT, but NMD670 is a groundbreaking small molecule designed to improve muscle function by targeting the ClC-1 chloride ion channel in skeletal muscles. For those living with CMT, this could mean better mobility, reduced fatigue, and an enhanced quality of life.
The drug is already in a Phase II clinical trial (SYNAPSE-CMT) to evaluate its safety and effectiveness for CMT types 1 and 2. The FDA’s recognition of NMD670 underscores its potential to meet critical unmet needs in our community, giving us hope for a brighter future.
This exciting progress shows that advancements in CMT research are happening, bringing us closer to treatments that could transform lives.
Interested in Joining the Clinical Trial?
By joining the study, you’ll play a critical role in determining whether this therapy could help others with CMT in the future.
NMD Pharma is currently recruiting eligible patients for the following sites:
NextGen Precision Health, Missouri
Principal Investigator: W. David Arnold, MD
Contact: Neetha Gali | Email: [email protected] | Phone: (573) 882-3065
National Neuromuscular Research Institute, Texas
Principal Investigator: Yessar Hussain, MD
Contact: Stephanie Gonsoulin, RN, BSN | Email: [email protected] | Phone: (512) 920-0140 x210
OSU Department of Neurology Division of Neuromuscular Diseases, Ohio
Principal Investigator: Bakri Elsheikh, MD
Contact: Lischele Watkins, MACPR | Email: [email protected]
Other locations, including those in New York, Boston, Kansas, Ohio, and Washington will be opening soon.
If you’re living with Charcot-Marie-Tooth (CMT) disease, specifically CMT1 or CMT2 subtypes, you may be eligible to participate in an exciting new clinical trial.
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