News

Soliris (eculizumab) may be the most effective and well-tolerated immunotherapy for patients with myasthenia gravis (MG), according to an analysis of multiple studies. The research, “Immunosuppressive and monoclonal antibody treatment for myasthenia gravis: A network meta‐analysis,” was published in the journal CNS Neuroscience…

Prograf (Tacrolimus) may be a beneficial treatment for people with myasthenia gravis (MG), helping reduce steroid use and disease scores, a joint analysis of several studies shows. The review, “Effectiveness and safety of tacrolimus therapy for myasthenia gravis: A single arm meta-analysis” was published in the…

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,…

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…