Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
Treatment with Descartes-08, an investigational CAR T-cell therapy, markedly eased symptoms of myasthenia gravis (MG) in three patients participating in a Phase 1/2 clinical trial assessing the therapy’s safety and preliminary efficacy. “This is the first clinical trial of CAR T-cells for an autoimmune disease. Naturally, the first patients…
For the first time, a genetic risk factor for myasthenia gravis (MG) has been identified that involves a gene — AGRN — coding for a protein directly involved in the activity of neuromuscular junctions (NMJs), which are impaired in MG, researchers report. The study, involving the genetic analysis of more than…
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
Differences in gut bacteria composition in people with myasthenia gravis (MG) may be a promising avenue to explore in developing new therapies, a study highlights. Findings suggest that these bacteria might contribute to disease onset and progression, its researchers wrote. The study, “Gut bacterial microbiota in…
An inflammation-associated protein called calprotectin is present at abnormally high levels in the blood of people with myasthenia gravis (MG), a new study reports. Data also suggest that levels of this protein might correlate with MG activity and severity, but further investigation is needed, according to its researchers. “There…
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
The European Medicines Agency (EMA) has agreed to review Argenx’s application requesting the approval of its lead candidate, efgartigimod (ARGX-113), to treat people with generalized myasthenia gravis (gMG). A regulatory decision is expected by mid-2022. “gMG is a severe, chronic and debilitating disease that can be unpredictable…
Myasthenia gravis (MG) tends to be more severe in people who are female, obese, or who took a long time to get diagnosed, according to a survey including nearly half of all MG patients living in Sweden. According to its researchers, nearly half of survey respondents reported having moderate…
One year of rituximab therapy was effective in people with myasthenia gravis (MG) ages 65 or older, with no significant differences in response or safety seen in this older patient group relative to younger patients, an analysis of medical records found. “Rituximab should be considered in the treatment…
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