In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar. William Whitman…
The case of a 55-year-old woman diagnosed with overlapping Lambert-Eaton myasthenic syndrome and myasthenia gravis — which tested negative for well-known neuromuscular junction antibodies — could help pave the way for further research into better, target-specific therapies, a study suggests. The study, “A Case of Triple-Negative Myasthenia…
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…
With about $18 million expected to be awarded in grants supporting research across neuromuscular diseases in 2020, the Muscular Dystrophy Association (MDA) continues to be a leader in efforts to better treat and otherwise ease life for those touched by Lambert-Eaton myasthenic syndrome (LEMS) as well…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.
Elevated levels of antibodies against certain proteins in the blood — SOX2, GABAb and N-type VGCC —  may help to more quickly identify small-cell lung cancer (SCLC) in people with Lambert-Eaton myasthenic syndrome (LEMS), a study suggests. The study “Neuronal antibody detection and improved lung cancer prediction in…
A case of Lambert-Eaton myasthenic syndrome (LEMS) that occurred secondary to lung cancer highlights the need for comprehensive antibody testing when doctors suspect that the immune system might be attacking the nervous system. The case was described in Thoracic Cancer, in the paper, “Anti-SOX1 antibody-positive paraneoplastic neurological syndrome…
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