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May 21, 2019May 21, 2019

CMT Association Shares Latest Disease Therapies, Research at Conference

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The Charcot-Marie-Tooth Association (CMTA) has granted $1 million to scientists searching for treatments, but it could cost up to $500 million to bring a viable disease therapy to market, according ... Read more

May 16, 2019May 16, 2019

#AANAM – Gene Therapy Treats CMTX Even After Disease Onsest, Mouse Study Suggests

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Gene therapy can be of benefit even after neuropathy has begun, research in a mouse model of X-linked Charcot-Marie-Tooth disease (CMTX) found. The study, “Gene therapy after onset of neuropathy provides ... Read more

May 15, 2019

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

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With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been ... Read more

May 14, 2019May 14, 2019

#AANAM — Combined Gene Therapy/RNA Strategy May Correct Cellular Changes in CMT2A, Study Suggests

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A combination of gene therapy and a strategy called RNA interference (RNAi) successfully repaired disease-related alterations in nerve cells derived from patients with Charcot-Marie-Tooth disease type 2A (CMT2A), according ... Read more

May 9, 2019May 9, 2019

#AANAM – Follow-up Study of DI-CMTC Patients Shows Progressive Nerve Damage, Gait Deterioration

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Patients with dominant intermediate Charcot-Marie-Tooth disease type C (DI-CMTC) experience progressive nerve damage, disability, and gait and quality of life deterioration, according to a follow-up study over 16 years. ... Read more

April 25, 2019April 25, 2019

Nonprofit Group Works to Raise Rare Disease Awareness in India

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With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients ... Read more

April 23, 2019April 23, 2019

New LRSAM1 Gene Mutations Found in French Families with CMT Type 2, Researchers Report

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Two new mutations in the LRSAM1 gene linked to Charcot-Marie-Tooth (CMT) disease type 2P were found in eight non-related French families, according to researchers. Their findings were published in the ... Read more

April 18, 2019April 18, 2019

CMTA Grants Thousands in Funding for STAR Research Projects

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The Charcot-Marie-Tooth Association (CMTA) has awarded approximately $1 million in grants to scientists worldwide investigating treatments and a cure for the disease of the peripheral muscle-controlling nerves. The grants ... Read more

April 16, 2019April 16, 2019

Further Research Needed to Understand Link Between Mutations in tRNA Enzymes and Charcot-Marie-Tooth Disease, Researchers Say

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Further studies are needed to understand how mutations in genes coding for aminoacyl-tRNA synthetases (aaRSs) may help fuel Charcot-Marie-Tooth (CMT) disease, researchers say. Their study, “Neurodegenerative Charcot-Marie-Tooth disease as a ... Read more

April 11, 2019April 11, 2019

Economic Burden of CMT Treatment Increases with Disease Severity, Age, German Study Reports

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Treatment of Charcot-Marie-Tooth (CMT) disease represents a substantial economic burden, which increases with age, disease severity, comorbidities, obesity and perceived impairments, according to a German study. The study, “Cost ... Read more

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