News

Gut Bacterial Markers May Aid Diagnosis of Children

Analysis of five bacterial markers present in the gut microbiome — the community of microbes living in the gut — may help in diagnosing myasthenia gravis (MG) in children, according to a Chinese study. The study, “Metagenome-wide association study of gut microbiome revealed potential microbial marker…

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…

Older Late-onset Patients Have Overall Positive Prognosis

Patients who start experiencing symptoms of myasthenia gravis (MG) after age 65 have an overall good prognosis and response to immunosuppressive treatments, according to a recent Canadian study. In addition, the presence of other medical conditions, or comorbidities, had no effect on the severity of MG symptoms or on…

Ultomiris Helps Ease gMG Symptoms in Ongoing Phase 3 Trial

Ultomiris (ravulizumab-cwvz) rapidly and sustainably eased symptom severity in adults with generalized myasthenia gravis (gMG), according to top-line data from a Phase 3 trial. Alexion Pharmaceuticals, the therapy’s developer, now plans to submit regulatory applications requesting approval of Ultomiris for gMG in the U.S., Europe, and Japan, later…

Nominations Open for 2022 Eurordis Black Pearl Awards

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

Standard MG Care Less Effective for Women, Danish Study Suggests

Women with myasthenia gravis (MG) given standard treatment improve significantly less in both objective and patient-reported outcome measures than do men, a study in Denmark with nearly five years of follow-up data reported. Notably, this sex difference was independent of age, disease duration, simultaneous health conditions (comorbidities), and prior…

Rare Case of MG Crisis Attributed to COVID-19 Vaccine

A very rare case of a myasthenia gravis (MG) crisis caused by the Moderna COVID-19 vaccine was described recently by researchers at St. Joseph’s Regional Medical Center in Paterson, New Jersey. “Although numerous studies have observed the relationship between active COVID infections and MG exacerbations, no known studies or…

Patient Dosing Begins in Phase 1/2a Trial of Oral NMD670

Patient dosing has begun in a Phase 1/2a clinical trial of NMD670, an investigational oral therapy by NMD Pharma aiming to improve motor abilities and quality of life for people with myasthenia gravis (MG). “We are pleased to announce the dosing of the first myasthenia gravis patient today…