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New research efforts by Neurogene, give hope to patients suffering with an ultra-rare type of CMT: CMT4J

New research efforts by Neurogene, give hope to patients suffering with an ultra-rare type of CMT: CMT4J

Jul 19, 2019 | CMT Research Updates, Research News

Neurogene, a leading company developing genetic medicines for people with rare, devastating neurological diseases, announced today that it is recruiting patients for a natural history study for CMT4J. “Key data now being collected to support endpoint assessment...

Is “Disease-In-A-Dish” in Combination with Animal Models A Faster Track to a Cure for CMT?

Jul 1, 2019 | CMT Research Updates, Research News

From time to time the leadership of CMTRF will explore the challenges of finding treatments and a cure for CMT.  To the extent we are able, we will try to overcome these obstacles to accelerate successful CMT research. Treatment of diseases, especially rare diseases...

Partnerships and Priorities: The Pathway to CMT Results

May 31, 2019 | CMT Research Updates, Foundation News, Research News

From time to time the CMT Research Foundation will examine the impediments to finding a cure for CMT.    As the Chairman of a nonprofit foundation whose ONLY mission is to fund research that will lead to a cure for Charcot-Marie-Tooth disease (CMT), I am often...

Where We Started, How Far We Have Come and Where We are Going

Nov 15, 2018 | Foundation News, Research News

Charcot-Marie-Tooth disease was identified in 1886. Yet, for the next hundred and twenty or so years there was no organized effort to help find an effective treatment, or better yet, a cure even though CMT affects one in 2,500 people (about the same prevalence as...
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