News

September 2, 2020September 2, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more

September 1, 2020September 1, 2020

AChR Autoantibodies Reflect Disease Severity, Study Indicates

News

Beyond being a diagnostic tool, antibodies against acetylcholine receptors (AChR) in the blood reflect disease severity and could be a useful biomarker for myasthenia gravis (MG) progression, a study ... Read more

August 28, 2020August 28, 2020

IMVT-1401 Seen to Safely Treat Generalized MG in Phase 2a Trial

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Treatment with IMVT-1401 significantly eased symptoms and lowered antibody levels in adults with moderate-to-severe generalized myasthenia gravis (MG), top-line results of a small Phase 2a trial show. Use of ... Read more

August 25, 2020August 25, 2020

IVIG Given Prior Prednisone Safely Prevents Most MG Flares, Study Finds

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Infusions of intravenous immunoglobulin (IVIG) given before high-dose prednisone safely prevents most of its associated exacerbations in people with generalized myasthenia gravis (MG), a study reports. The study, “Intravenous immunoglobulins may ... Read more

August 24, 2020August 24, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

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Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient ... Read more

August 21, 2020August 21, 2020

Johnson & Johnson to Acquire Momenta and Rights to Nipocalimab

News

Johnson & Johnson will acquire Momenta Pharmaceuticals, which has been developing nipocalimab (M281) for the treatment of myasthenia gravis (MG). The process, which involves an all-cash deal totaling about ... Read more

August 18, 2020August 18, 2020

Levels of suPAR Protein Linked to Myasthenia Gravis Severity

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Measuring levels of a protein known as suPAR in the blood could be useful for assessing the severity of myasthenia gravis (MG), a pilot study reports. The study, “Serum ... Read more

August 17, 2020August 17, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

News, syndicated

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. ... Read more

August 14, 2020August 14, 2020

Firdapse Falls Short in Phase 3 Trial in MuSK-MG Patients, Top-line Results Show

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Firdapse (amifampridine phosphate), an investigational treatment for MuSK-antibody positive myasthenia gravis (MuSK-MG), failed to meet its primary or secondary goals, according to top-line results from a Phase 3 clinical ... Read more

August 11, 2020August 11, 2020

Rituximab Effectively Treats Refractory Juvenile MG Patients, Small Study Suggests

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Rituximab, an approved treatment for certain cancers and autoimmune conditions, appears to also be effective and well tolerated in people with juvenile myasthenia gravis (JMG) who fail to respond ... Read more

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