News

April 24, 2020April 24, 2020

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

News, syndicated

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. ... Read more

April 17, 2020April 17, 2020

Extended Protein Form Linked with Myelin Damage, May Underlie CMT, Mouse Study Finds

News

A larger-than-normal version of myelin protein zero is associated with damage to myelin — the protective sheath covering nerve fibers — and may underlie the development of Charcot–Marie–Tooth (CMT) disease, ... Read more

April 10, 2020April 10, 2020

Nerve Conduction Changes Not Linked to Quality of Life and Hand Function in CMT1A Patients, Study Says

News

Muscle wasting and nerve conduction deficits are not associated with alterations in health-related quality of life (HRQoL) and hand function in people with Charcot-Marie-Tooth disease type 1A (CMT1A), a ... Read more

April 7, 2020April 7, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

News, syndicated

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most ... Read more

April 3, 2020April 3, 2020

With $30M Investment, Regenacy Plans Phase 2 Trial Testing Ricolinostat for CMT Type 2

News

Regenacy Pharmaceuticals received $30 million to help advance its lead candidate ricolinostat into Phase 2 studies for Charcot-Marie-Tooth (CMT) disease type 2, as well as diabetic and chemotherapy-induced peripheral ... Read more

March 31, 2020March 31, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

News

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. ... Read more

March 27, 2020March 27, 2020

PXT3003 Designated Promising Innovative Medicine in UK for CMT1A Treatment

News

The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) designation to PXT3003 for the treatment of people with Charcot-Marie-Tooth type 1A (CMT1A) aged 16 and older. PIM ... Read more

March 20, 2020March 20, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

News

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see ... Read more

March 20, 2020March 27, 2020

Pharnext Raises 7.7M Euros to Develop PXT3003 Therapy for CMT1A

News

France-based Pharnext will use a capital raise of about 7.7 million euros ($8.4 million) to support the clinical development of PXT3003, the company’s experimental treatment for Charcot-Marie-Tooth disease type ... Read more

March 17, 2020March 17, 2020

Mutation in SEPT9 Gene Can Lead to CMT, Report Says

News

A mutation in the SEPT9 gene — which provides instructions for making the septin-9 protein that is involved in many cell functions, including cell division and movement — can ... Read more

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