News

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term impact…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

Women with myasthenia gravis can safely undergo normal vaginal delivery and avoid a cesarean section with the help of epidural labor analgesia (ELA) to minimize fatigue associated with childbirth, a study says. The findings of the study, “Management of labor and delivery in myasthenia gravis:…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

A vast majority of patients with myasthenia gravis (MG) responded to treatment with plasma exchange and half experienced symptom relief, according to a study. The findings also showed that men and patients with late-onset MG were more likely to have successful response to the treatment. The study, “…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

Treatment with Soliris (eculizumab) provided adults with generalized myasthenia gravis (gMG) clinically meaningful improvements through three years in daily living, muscle strength, functional ability and quality of life, according to early data from an extension study. Lower rates of MG exacerbations and hospitalizations were also observed. The…

Treatment of juvenile myasthenia gravis (JMG) lessens the severity of symptoms in most patients, leading to stable remission without significant amounts of medication, according to a study of Polish patients. The study, “Treatment outcome in Juvenile-onset Myasthenia Gravis,” appeared in the journal Muscle & Nerve.

A small regulatory RNA, called microRNA-653, inhibited the proliferation of thymus cells, called thymocytes, in a mouse model of myasthenia gravis (MG), a study reports. Given that the thymus is often abnormally enlarged in MG patients, these results, although preliminary, suggest that microRNA-653 may be a potential therapeutic…