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October 19, 2018October 19, 2018

International CMT&Me Observational Study to Explore the Impact of CMT Using an App

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A newly launched international observational study is going to explore the overall impact of Charcot-Marie-Tooth disease (CMT) using a tailored app called CMT&Me. Designed by Vitaccess in collaboration with ... Read more

October 17, 2018October 17, 2018

PXT3003 Eases Disability, Found Safe in CMT1A Patients, Phase 3 Trial Shows

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Investigational compound PXT3003 consistently eased disability in patients with mild to moderate Charcot-Marie-Tooth disease type 1A (CMT1A), according to top-line Phase 3 results from Pharnext, which also found that the ... Read more

October 15, 2018

Hereditary Neuropathy Foundation Hosts FDA Patient-Focused Drug Development Meeting

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Along with Chris Comish, President and CEO of Charcot-Marie-Tooth’s parent company BioNews Services, I recently had the opportunity to attend the FDA Patient-Focused Drug Development (PFDD) Meeting for Charcot-Marie-Tooth ... Read more

October 12, 2018October 12, 2018

Sarepta Acquires Rights to CMT Gene Therapy Program from Nationwide Children’s Hospital

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Nationwide Children’s Hospital (NCH) has granted the exclusive rights of neutotrophin 3 (NT-3), its gene therapy candidate for the treatment of Charcot-Marie-Tooth (CMT) neuropathies, to Sarepta Therapeutics. Researchers plan ... Read more

October 10, 2018October 10, 2018

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

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Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based ... Read more

October 10, 2018October 10, 2018

CMT Research Foundation to Fund Search for Treatments and Cures

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Two Charcot-Marie-Tooth (CMT) patients, Patrick Livney and Susan Ruediger, have launched the CMT Research Foundation (CMTRF), a nonprofit group committed to developing treatments and cures for the disease. The ... Read more

October 8, 2018October 8, 2018

CMTX5 in Brothers Marked by Temporary Muscle Weakness After Fever, Case Report Says

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Two brothers with X-linked Charcot-Marie-Tooth disease type 5 (CMTX5), who carried newly identified mutations in the PRPS1 gene, experienced temporary but recurrent symptoms of muscle weakness after taking ill with ... Read more

September 21, 2018

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

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More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products ... Read more

September 21, 2018September 21, 2018

HNF Launches Improved Online Survey, Hosts CMT Health Summit

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In order to advance research to improve medical treatment and care for those living with Inherited Neuropathies (IN), such as Charcot-Marie-Tooth (CMT), the Hereditary Neuropathy Foundation (HNF) is relaunching ... Read more

September 19, 2018September 19, 2018

Implanting Stem-derived Schwann Cells Improves Motor Activity, Nerve Regeneration in CMT1A

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Implanting stem-derived Schwann-like cells into mice with Charcot-Marie-Tooth type 1A improved neuron function, leading to better motor activity and nerve regeneration, according to new research from South Korea. The ... Read more

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