A newly reported case from Nanchang University suggests that efgartigimod, a neonatal Fc receptor (FcRn) blocker approved for generalized myasthenia gravis, may offer a promising treatment option for patients who…
I was diagnosed with myasthenia gravis, or MG, in 2021 when I was in my early 20s. My journey began at a friend’s birthday party when I noticed my face…
I've always known that I wanted to help people. Growing up as the son of a veterinarian and a high school teacher who later became a physician assistant, all with…
A new RNA-based drug from Regeneron Pharmaceuticals and Alnylam Pharmaceuticals has shown positive results in a late-stage clinical trial for generalized myasthenia gravis (gMG), a rare, chronic autoimmune disease that…
In our second installment from our series of reports provided by our partner TREND Community, we are going to be focusing on a different condition over the next four weeks. …
In 2017, Jamie Shultz was feeling on top of the world. She was 25 years old, engaged to the man of her dreams, had just passed the Bar exam, and…
In an inspiring act of love and advocacy, Joburg resident Nthabiseng Mokoena has completed a remarkable 35-day walk from Johannesburg to Cape Town to raise awareness for a rare disease…
The holidays are a time of fun, family, and for many of us, a time when our chronic health conditions tend to flare up. We don’t mean for that to…
Written by Audrey Getman September 25, 2020: The day I was diagnosed with myasthenia gravis (MG). One month prior: I was in the emergency room (ER) after experiencing a worsening…
By Alexis Rodriguez When I was 24 years old, what started as a terrible sinus infection took a turn I never could have expected. I woke up one morning with…
Cartesian Therapeutics, Inc., a biotechnology company, recently announced that the FDA has awarded the Regenerative Medicine Advanced Therapy (RMAT) designation to its lead product, Descartes-08, to treat myasthenia gravis (MG).…
While there is no cure for myasthenia gravis, this disorder can be managed with treatment. Current treatment options include a thymectomy, C5 protein inhibitors, and intravenous immune globulins (among others).…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Biomarkers are objective measures that scientists, researchers, and doctors can use to understand what is going on in your body. For example, your blood pressure can tell doctors about your…
The holidays are a time of fun, family, and for many of us, a time when our chronic health conditions tend to flare up. We don’t mean for that to…
The biotechnology company Cabaletta Bio has recently announced that the company investigational new drug (IND) application for its experimental CD19-CAR T-cell therapy CABA-201 has been cleared by the US Food…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
The FDA has approved Rystiggo, a developed by UCB in Brussels, to treat two types of myasthenia gravis. The approval was based on the results of the Phase III…
In late June 2023, global biopharmaceutical company UCB announced that the U.S. Food and Drug Administration (FDA) approved RYSTIGGO (rozanolixizumab-noli) for adults living with AchR or MuSK antibody positive…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
In a recent news release from biotechnology company Cartesian Therapeutics, the company shared that the first patient was dosed in a Phase 2b study evaluating Descartes-08 for generalized myasthenia…
Mazidul Islam told the Daily Star that he had to watch his 14-year-old son Farid die and could do nothing to save him. Among other things, he regrets the fact…
In a mid-November 2022 press release from biopharmaceutical company CANbridge Pharmaceuticals, Inc. (“CANbridge”), the company shared that its therapeutic option CAN106, which binds to and neutralizes C5 in the…
In April 2022, the United States approved Ultomiris for the treatment of patients with generalized myasthenia gravis (gMG). This approval was followed, just four months later, by an approval…
According to a story from Myasthenia Gravis News, the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) has recommended ravulizumab (marketed as Ultomiris) to be approved as…
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