On Thursday, April 10, more than 120 patients, scientists, clinicians and pharma companies gathered in Columbus, Ohio for OHio, What a Night — an unforgettable evening dedicated to accelerating research and developing treatments and cures for Charcot-Marie-Tooth disease.

OHio, What a Night highlighted the groundbreaking CMT Gene Therapy research taking place in Columbus — home to Nationwide Children’s Hospital and The Ohio State University — and raised more than $110,000, which will be used to fund CMT research.

The evening’s highlight was a keynote presentation by Dr. Jerry Mendell, a leading gene therapy pioneer, and an advisor to the Jerry R. Mendell Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and Sarepta Therapeutics. Dr. Mendell shared his vision for how Columbus — where FDA-approved gene therapies for Duchenne muscular dystrophy and spinal muscular atrophy were developed — could be the birthplace of the first gene therapy for CMT. “CMT is ready,” Dr. Mendell said, inspiring a room full of CMT researchers, clinicians and patients and their families with hope for what’s next.

Fueled by the support from the event sponsors and host committee, OHio, What a Night spotlighted how far CMT research has come — and the collaborative spirit driving it forward.

CMT Research Foundation is proud to be funding three CMT gene therapy projects at Nationwide Children’s Hospital, focused on CMT1A (in collaboration with Armatus Bio), CMT1B and the development of next-generation delivery vehicles to make gene therapies more effective across many types of CMT.

On Friday, April 11, members of the CMT community had the opportunity to tour the labs of Dr. Afrooz Rashnonejad, Dr. Zarife Sahenk and Dr. Scott Harper at Nationwide Children’s Hospital, where these cutting-edge projects are underway. It was powerful to witness the very place where breakthroughs for other neuromuscular diseases began — and where CMT is poised to be next.

Thank you to our sponsors who made this event possible:

Presenting Sponsors

• Nanette Nardi Triplett and Michael Triplett, Ph.D.

Full Moon Sponsors

• Zarife Sahenk, MD, Ph.D.
• The Stahl Family

Crescent Moon Sponsors

• Andelyn Biosciences
• Armatus Bio
• Nationwide Children’s Hospital
• Neucore Bio
• NMD Pharma
• Ohio Life Sciences
• The Ohio State University, Wexner Medical Center
• Sarepta Therapeutics

Twilight Sponsors

• CK Contruction
• Cleary and George Simpson
• Danis
• LaBovick Law Group
• Mr. and Mrs. Kenneth Morand
• Rachel Salzman and Michael Weinberg
• Werth

Special thanks to our host committee: Michael Triplett, Nanette Nardi Triplett, Kenny Morand and Rachel Salzman of Armatus Bio — as well as all the researchers and partners from Nationwide Children’s Hospital, The Ohio State University, and beyond who are helping us light the path to a cure.

To watch a recording of the evening, visit youtu.be/KOmF4pTYkB0