According to a recent press release from XBiotech, a Texas-based biotechnology company, patient dosing is now underway in a phase 1 clinical study of the Company's investigational systemic sclerosis treatment, bermekimab.…
According to a story from news-medical.net, the National Institutes of Health (NIH) has doled a total of $31 million on grants this year to 20 different research teams. The goal?…
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A recent article in Forbes highlights a study on diagnostic performance published in The Lancet Digital Health. The study represents the first review to compare the diagnostic accuracy of deep…
According to a story from Medical News Bulletin, a recent study that was first published in the New England Journal of Medicine appears to have revealed a promising new treatment for ulcerative…
According to a story from med.miami.edu, an international research consortium known as Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) has just been awarded a renewal grant from…
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Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…
According to a story from Ankylosing Spondylitis News, a recent study has found that moxibustion, a form of heat therapy, could provide short-term benefits for patients with the rare disease…
A recent press release from Fulcrum Therapeutics, a Massachusetts-based biotechnology company, announced preliminary findings of the company's phase 1 clinical study of losmapimod in the treatment of facioscapulohumeral dystrophy. Losmapimod is…
According to a story from BioSpace, a global collaborative called the Trajectory Analysis Project recently released a study that utilized the North Star Ambulatory Assessment (NSAA), a recently developed measurement…
According to a story from ncbiotech.org, a new partnership between Vertex Pharmaceuticals and RNA-based therapeutics company Ribometrix aims to develop potential treatments for serious diseases, including Huntington's disease, which is…
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According to a recent article published by the University of California San Francisco, Pelizaeus-Merzbacher disease (PMD) is a rare neurological disease affecting young boys. PMD fatalities may occur before the child…
According to a recent press release from the Massachusetts-based biotechnology company Avrobio, Inc., the first patient has been dosed in the Company's phase I/II clinical study of their investigational cystinosis…
A Phase 3 trial for Cushing syndrome led by Maria Fleseriu at Oregon Health and Science University has just been completed. It examined a new investigational treatment called levoketoconazole. About the…
Gene therapies have huge potential in the field of rare disease. The fact that these therapies could offer a potential cure otherwise fatal diseases has given families incredible hope. Unfortunately,…
According to a story from the 23andMe Blog, Reni Winter-Evans, who was diagnosed with Parkinson's disease, understands that you don't have to be a scientist to be a big part…
Geisinger Health System is based out of Pennsylvania. They have recently initiated a novel program that has goals to make DNA sequencing a standard part of routine clinical care. Next…
According to a story from news-medical.net, a team of researchers have developed a potential new treatment for polycystic kidney disease (PKD), a rare disorder that ultimately causes organ failure. The…
According to a story from pmlive.com, the gene therapy company Bluebird Bio's latest recently released results from the company's phase 2/3 clinical trial demonstrates further encouraging data for its Lenti-D…
NASH Nonalcoholic Steatohepatitis (NASH) is a rare disease in which hepatic inflammation and injury to the cells occur as a result of steatosis (hepatic fat accumulation). Severe cases of NASH…
Prevalence of MS in Korea In South Korea, rates of multiple sclerosis (MS) are generally low. This is because the disease is most prevalent in areas where there is lower sun…
As originally reported in Cambridge Independent, inflammatory bowel diseases are about to get a boost in research after Health Data Research UK has awarded a £5 million grant to a…
With advocacy organizations gaining traction, sometimes patients with rare diseases may have a handful of options to choose from. Some patients may be quick to join patient advocacy groups…
The Bad News Unfortunately, it has recently been announced that an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD) will not be continuing on in clinical trials. This is because the…
Innovation and Collaboration Macular telangiectasia type 2 (MacTel) is a rare eye disease that causes progressive vision loss as patients age. Paul Bernstein is a physician at the John A.…
According to a story from Fabry Disease News, the first patient in a clinical trial testing the investigational gene therapy FLT190 has reportedly been dosed. FLT190 is being developed as…
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