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Since March, most of the globe has been anxiously awaiting updates on COVID-19, the virus that has infected over 74 million people. One update was just released: the CDC has…
Target ALS has recently launched a new research initiative, in which they will dedicate $15 million to amyotrophic lateral sclerosis (ALS) research, specifically the discovery of biomarkers. These millions will…
Dan Hopkins is a bowler who has recently raised over $21,000 for cystic fibrosis (CF) in a 65 hour long bowl-a-thon. While he may be exhausted and sore, he is…
Crystal Goodwin, from South Portland, Maine, was diagnosed with mast cell activation syndrome (MCAS) back in 2011. A rare disease diagnosis can turn one's life upside down, and Crystal is…
Xencor, a biopharmaceutical company that focuses on curating treatments for cancers and autoimmune conditions, has recently released data on vibecotamab. This acute myeloid leukemia treatment was included in a Phase…
As of last month, a newborn screening advisory committee from the Virginia Department of Health declined to add Krabbe disease to the conditions that doctors look for when a child…
Rare disease patients often face a long, complicated, and frustrating journey to the right diagnosis and treatment. Ally Trainor, a 21-year-old student at George Mason, is no exception. She has…
The Yale Cancer Center has recently presented the findings of their study of the cost-effectiveness of caplacizumab at the 62nc ASH Annual Meeting and Exposition. Their conclusion, which is that…
A report from the Neurological Alliance calls for improvements in treatment and care for the 150,000 rare neurological disease patients in the UK, including spinal muscular atrophy (SMA) patients. It…
As of November 20, Zokinvy has been approved by the FDA for the treatment of Hutchinson-Gilford progeria syndrome (progeria) and a few other progeroid laminopathies in patients aged one year…
According to a story in Charcot-Marie-Tooth News, the CMT&Me study has launched a competition in Europe with the purpose of raising awareness and money for CMT and related disorders. Any…
A review of COVID-19 data from the UK shows that patients with rare autoimmune rheumatic disease (RAIRD) face a higher mortality risk, according to an article in Healio. The study,…
A study in the European Medical Journal aims to confirm the safety profile of vedolizumab, a treatment for inflammatory bowel disease (IBD). Specifically, they focused on acute infusion reactions. It…
The FDA has recently granted its Rare Pediatric Disease designation to Moleculin's WP1066 for three indications: atypical teratoid rhabdoid tumor, diffuse intrinsic pontine glioma, and medulloblastoma. This designation will aid…
While there is still a lot left to learn about COVID-19, there are a number of facts that we know, one of which is that children face less severe cases.…
Mission: Cure is a coalition of patients, researchers, doctors, and entrepreneurs who have taken a new approach to curing diseases. Their first focus is pancreatitis, and they have created a…
Part of the National Institutes of Health (NIH), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and SteroTherapeutics have entered into a Cooperative Research and…
A study published in Neurology has discovered a way to improve autoantibody testing in myasthenia gravis (MG) patients. An issue that physicians and patients face is false-positive results for neuromuscular or autoimmune…
Gut bacteria has been known to play a role in a number of conditions, such as inflammatory bowel disease, rheumatoid arthritis, and more. A new study has discovered a connection…
The FDA has recently approved DANYELZA for the treatment of neuroblastoma in pediatric patients over the age of one and adult patients affected by refractory or relapsed neuroblastoma. This approval…
Prevail Therapeutics, a company that focuses on developing gene therapies for neurodegenerative diseases, has announced that a composition of matter patent has been issued for PR001. This therapy is intended…
There is a stigma that comes with the term "palliative care," as it is typically associated with hospice and terminal illnesses. This is not the case, as palliative care is…
Maintaining lung function is a major goal of cystic fibrosis (CF) treatment. In order to accomplish this goal, doctors will use airway clearance techniques, such as vest therapy. The problem…
Innovent Biologics has recently received approval from the National Medical Products Administration (NMPA) for their juvenile idiopathic arthritis (JIA) treatment, SULINNO. It has been approved to treat JIA, rheumatoid arthritis,…
Takeda Pharmaceuticals has recently awarded XOMA Corporation $2 million for reaching the next milestone in the development of mezagitamab, a treatment for myasthenia gravis (MG). According to Biospace, XOMA has…
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