The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…
News
Local injections of the corticosteroid dexamethasone alleviated symptoms and resulted in complete remission in most patients with ocular myasthenia gravis, a small clinical trial has shown. Compared to systemic treatments, local injections treated symptoms faster, reduced side effects, and increased remission rate.
An analysis of the thymuses removed from people with myasthenia gravis (MG) found lower levels of micro RNA molecules (miRNAs) that may contribute to disease development by favoring increased production of pro-inflammatory proteins and cells, a study suggests. …
Argenx is looking for participants in an app-based international study of the impact and burden of myasthenia gravis (MG) on patients and their families. The company has released an app called MyRealWorld MG to gather information about individuals’ diagnosis, complications, treatments, activities, and quality of life, using…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,” the…
A Japanese man with generalized myasthenia gravis (gMG) experienced a severe worsening of symptoms after discontinuing treatment with Soliris (eculizumab), a case study reported. Titled “Severe worsening of myasthenic symptoms after…
Activating the bile acid receptor TGR5 was found to reduce the number of immune cells that secrete proinflammatory proteins, suggesting that this receptor may be a therapeutic target for people with myasthenia gravis (MG), a study reported. The study, “…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
The first published case of a person developing ocular myasthenia gravis (MG) secondary to COVID-19 was described in a new report. The patient, a 65-year-old woman with a medical history of kidney cancer, pituitary tumor, and a blockage in the pulmonary artery in the lungs, ultimately was successfully…
The first participant has been dosed in a Phase 1/2a trial evaluating NMD670, NMD Pharma‘s investigational therapy for myasthenia gravis (MG). “I am pleased to announce that our lead program has entered the clinic. NMD670 has already shown great promise in preclinical studies and…
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