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Research News

Importance of Biomarkers in CMT

Jan 21, 2020 | CMT Research Updates, Research News

Using Biomarkers to Measure Effective Treatment Options for CMT

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Targeting Axon Degeneration – a treatment for all types of CMT?

Jan 7, 2020 | CMT Research Updates, Research News

Preventing axon degeneration could stop the progression of CMT. Read about the efforts by pharma to stop axon degeneration.

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Are We Prepared for Clinical Trials in Charcot-Marie-Tooth?

Jan 7, 2020 | CMT Research Updates, Research News

A Review of Clinical Trial Readiness for CMT BY CMT RESEARCH FOUNDATION'S SCIENTIFIC ADVISORY BOARD MEMBER DR. ALEXANDER ROSSOR Read the full paper In this issue of Brain Research, Mike Shy, Mary Reilly and I summarize the current array of outcome measures for...

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Partnership with DTx Pharma to optimize antisense oligonucleotides as a gene therapy for CMT1A

Partnership with DTx Pharma to optimize antisense oligonucleotides as a gene therapy for CMT1A

Dec 20, 2019 | CMT Research Updates, CMTRF Funded Research, Research News

ATLANTA (December 20, 2019) The CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth, today announced it has partnered with DTx Pharma, an RNA medicines company breaking open new therapeutic areas for...

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A Robust Therapeutic Pipeline for CMT 1A

A Robust Therapeutic Pipeline for CMT 1A

Dec 19, 2019 | CMT Research Updates, CMTRF Funded Research, Research News

The majority of people with CMT have the 1A subtype which is due to duplication of the peripheral myelin protein 22 (PMP22) gene leading to breakdown of the myelin that surrounds peripheral nerves.  With time this loss of myelin causes nerve dysfunction and...

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Drug Screening in a Dish

Drug Screening in a Dish

Dec 6, 2019 | CMT Research Updates, CMTRF Funded Research, Research News

At the CMT Research Foundation we are very much interested in using patient-derived stem cells called iPSCs for drug screening in order to develop therapies for CMT. Indeed, one of the first projects we funded was such a drug screening effort for CMT2A at Acurastem....

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Gene Editing Used for the First Time in CMT

Gene Editing Used for the First Time in CMT

Nov 22, 2019 | CMT Research Updates, CMTRF Funded Research, Research News

Gene editing as a therapeutic tool for treating disease has been very much in the news recently.  The most commonly used gene editing tool to permanently fix the underlying genetic cause of a disease is CRISPR/Cas9. A recent paper demonstrates the first...

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Chief Science Officer comments on recent work

Chief Science Officer comments on recent work

Nov 11, 2019 | CMT Research Updates, Foundation News, Research News

CMT Research Foundation Chief Science Officer Grace Pavlath, PhD comments on recent work by Morelli et al: Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models Some types of CMT result from the expression of a mutant...

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Emerging Landscape of Modifier Genes in CMT

Oct 21, 2019 | CMT Research Updates, Research News

In CMT, there is a large variation in disease onset, severity, or progression, often seen among individuals with the same genetic subtype of a disease, even within a single family.  Genetic modifiers play important roles in such variations, known as heterogeneity....

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Inherited Neuropathy Consortium Awarded $7.2M to Continue Studying, Improve Care for CMT

Oct 11, 2019 | CMT Research Updates, Research News

The National Institutes of Health has awarded the Inherited Neuropathies Consortium (INC) $7.2M over the next five years. This grant will be focused on improving clinical care and clinical research for CMT. The CMT Research Foundation is a proud partner of the INC....

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