News

The Japanese Patent Office (JPO) has issued a new patent for the composition of eculizumab, marketed as Soliris by Alexion Pharmaceuticals, and its pharmaceutical formulations until 2027. Alexion is currently pursuing a patent for additional indications of Soliris, including for the treatment of anti-acetylcholine receptor (AchR) antibody-positive generalized…

Vaccination for protection against tetanus is safe and does not aggravate any clinical or immunological features of myasthenia gravis, according to the results of a report published in the journal Vaccine. Myasthenia gravis is an autoimmune disorder caused by the abnormal production of antibodies that attack one’s body. These…

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…

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…

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…