News

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

People who develop ocular myasthenia gravis (OMG) before age 40 are more likely to go into remission after surgery to remove the thymus (thymectomy), according to a Chinese study. The work also supported thymectomy as a safe and effective approach to treat OMG, with about 40% of patients…

Treatment with rituximab is safe, provides clinical improvements and enables stopping maintenance therapies in patients with late-onset myasthenia gravis (LOMG), a small study suggests. The study, “Rituximab in Late‐Onset Myasthenia Gravis is Safe and Effective,” was published in the journal Muscle & Nerve. Rituximab —…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

Injections of botulinum toxin type A — commonly known as Botox — for cosmetic or medical purposes were linked to transient side effects similar to symptoms of myasthenia gravis (MG) in two people detailed in a case report. The study, “Botulinum toxin injections associated with suspected myasthenia gravis: An…

FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

New guidelines have been published to help patients and physicians manage myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) during the COVID-19 pandemic. Broadly, the guidelines suggest that people with MG or LEMS should continue on their current therapies and take extra precautions to minimize the chance…

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. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…