Geisinger was founded by Abigail Geisinger over 100 years ago. It now has 13 hospitals and 2 research centers. They just released a new study examining the severity of symptoms…
In an article from the Orphanet Journal of Rare Diseases, a recent study provides an overview of the treatment of familial Mediterranean fever (FMF) that does not respond to the standard…
According to a story from MedicalXpress, the results from a recent study are linking biochemical changes affecting a protein known as CHIP to a diverse array of clinical characteristics that…
According to a posting from clinicaltrials.gov, an observational study will be focused on the identification of biomarkers for pediatric congenital heart disease and pulmonary arterial hypertension. Biomarkers derived from the…
According to a story from the US Food and Drug Administration (FDA), Dr. Amy Abernethy, who is the current Prinicipal Deputy Commissioner at the agency, was eager to start working…
According to a story from PR Newswire, the National Institutes of Health (NIH) and Three Lakes Partners have awarded a grant to the tune of $22 million to the Pulmonary…
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Stéphanie Tomé is an investigator at the Sorbonne Université in Paris, France. A recent article in PacBio describes Tomé’s research into a disease that becomes progressively worse with each generation.…
According to a story from Parkinson's News Today, the results of a global, online survey appear to indicate that genetically based, inherited Parkinson's disease is actually more common than the…
Elena Frid is neurologist and Lyme disease specialist. She lives in New York City. After opening her neurology practice in Manhattan, she quickly became aware of some huge issues many…
According to a story from WBUR, a preliminary evaluation of a young patients diagnosed with Tay-Sachs disease appears to show some encouraging signs. Two patients were treated with an experimental…
Firdapse, or amifampridine phosphate, is a medication currently approved for Lambert-Eaton myasthenic syndrome, or LEMS. However, Catalyst Pharmaceuticals has been working to evaluate if it can aid other neurological and…
According to a story from Medscape, the drug burosumab (marketed as Crysvita) demonstrated its ability to provide long term benefits for nearly two years to patients with the rare disease…
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A Colombian family with more than six thousand living family members was the focus of researchers at several institutes in the U.S. and Columbia. According to an article published by…
Data in the Modern Age The depth of information available today is unique to our age of technology. Current advances provide a platform to consolidate information on a wide array…
According to a story from Medical Xpress, a team of researchers affiliated with the University of Granada and the research organization CIBERFES have discovered a pair of new mutations linked…
According to a story from scienceblog.com, a recent study has revealed that large portion of cystic fibrosis cases recorded in the Dominican Republic and Puerto Rico are linked to genetic…
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The concept of N-of-1, or personalization of clinical care, originated in the 1980s according to an article in The Scientist. It began with a sixty-five-year-old Ontario man who was…
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490 words 8% vs 1306 words 3% According to a recent Globe Newswire release, Progenics Pharmaceuticals, an oncology company that specializes in artificial intelligence and targeted therapies, announced results for its Phase…
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According to a recent article in Biospace, Catalyst Pharmaceutical’s CMS-001 Phase 3 study ended with mixed results. The trial was billed as the first placebo-controlled double-blind study testing amifampridine phosphate (brand…
According to a story from us.acrofan.com, the biopharmaceutical company AstraZeneca recently announced the release of positive results for a clinical trial testing the company's drug durvalumab (marketed as IMFINZI) in…
"We went from being able to treat no patients to being able to treat nearly all patients in seven years." 90% of cystic fibrosis (CF) patients now have a new treatment…
According to a story from Medical Xpress, an international team of scientist from 34 different countries are putting their heads together to identify the most effective treatments for ulcerative colitis,…
FA Fanconi anemia (FA) is a rare disease in which the bone marrow in the body essentially fails. It is a genetic condition where the body progressively becomes deficient in…
GlaxoSmithKline (GSK) has just announced that Zejula, otherwise known as niraparib, has just been approved for cancer patients beyond those who have a BRCA+ mutation. The patients now included in…
According to a story from Fabry Disease News, a recent study has found that certain cells tend to age more rapidly in patients with Fabry disease when compared to unaffected…
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