News

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

Ancora Innovation is funding a research project at Vanderbilt University aiming to realize potential new treatments for Charcot-Marie-Tooth disease (CMT), according to a university press release. The research will be led by Charles Sanders, PhD, a biochemistry professor and associate dean for research at Vanderbilt. Sanders and his…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

AcuraStem has identified 37 compounds that improve nerve cell survival and might help treat Charcot-Marie-Tooth disease type 2A (CMT2A), according to a press release from the CMT Research Foundation. CMT2 accounts for roughly one-third of CMT cases and CMT2A is its most common subtype. Inherited mutations in…

As a result of significant reductions in walking and physical therapy, people with Charcot-Marie-Tooth (CMT) disease reported worsening physical skills and pain scores after the COVID-19 lockdowns, a recent survey found. Their responses highlight the need to encourage CMT patients to resume their previous…

An international observational study using a smartphone-based app called CMT&Me is exploring the impact of Charcot-Marie-Tooth disease (CMT) on patients and caregivers. The study (NCT03782883) was designed by Vitaccess in collaboration with Pharnext. Its goal is to collect detailed patient-reported data to help researchers and clinicians…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

Functional surgery can improve walking ability in people with Charcot-Marie-Tooth disease (CMT), and patients are generally satisfied with the outcomes, a study indicates. The study, “Long‐term walking ability and patient satisfaction after lower limb functional surgery in patients affected by charcot‐marie‐tooth disease: A retrospective study,” was published…

The CMT Research Foundation has launched a new research program that will seek to develop a precision medicine approach to treat people with Charcot-Marie-Tooth (CMT) disease, the foundation announced in a press release. The approach focuses on silencing the gene that is causing the disease in each…

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…