Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…
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The first participant has been dosed in a Phase 1 clinical trial evaluating argenx‘s subcutaneous formulation of ARGX-113 in healthy volunteers. ARGX-113 is an investigational antibody designed for the treatment of patients with severe autoimmune diseases associated with high levels of harmful immunoglobulin G (IgG) antibodies, such as myasthenia gravis (MG).
The risk of aggravating myasthenia gravis (MG) is higher in patients who develop influenza-like illness (ILI) than those who have been vaccinated against influenza vaccination, indicating that people with MG should be immunized, a study finds. The study, “The Impacts of Influenza Infection and Vaccination on Exacerbation of Myasthenia…
The U.S. Food and Drug Administration has approved Soliris (eculizumab) as a treatment for adults with a particular type of generalized myasthenia gravis (gMG) — the first time in more than 60 years that it has authorized a therapy for the disease. Alexion Pharmaceuticals, which developed Solaris, said the…
Researchers have juvenile myasthenia gravis (MG) to a specific variant, or allele, of the HLA gene among Norwegians. Their study, “Juvenile myasthenia gravis in Norway: HLA-DRB1*04:04 is positively associated with prepubertal onset,” appeared in the journal PLOS ONE. The disease mechanism that causes MG is complex, as it…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
Alteration of eye movement due to muscle weakness is not uncommon among patients with myasthenia gravis (MG). But now researchers at University of Cape Town (UCT) have identified the main characteristics of patients with juvenile-onset MG and of African genetic ancestry who present ocular manifestations associated with MG and do…
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#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
Myasthenic crisis is a common complication in patients with myasthenia gravis (MG), marked by exacerbation of MG symptoms and respiratory failure. But with the rarity of MG, and its similarity to a range of other neuromuscular diseases, proper detection and treatment might fail, researchers argue. To make it easier for…
The U.S. Food and Drug Administration has granted orphan drug status to ARGX-113, a potential treatment for myasthenia gravis, its developer argenx said in a press release. MG is an autoimmune disease triggered by immunoglobulin G auto-antibodies, or IgGs. They attack critical signaling proteins at the junction between nerve and…
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