An article from Comic Sands recently covered the story of a UK mother, Alison Beesley, who is now committed to taking care of her daughters Eliza and Eleanor full time. Eliza is nine years old and…
According to a story from globenewswire.com, the biopharmaceutical company Mustang Bio, Inc. has recently announced that the US Food and Drug Administration (FDA) has recently awarded the company Orphan Drug…
According to a story from drugs.com, the pharmaceutical company Pfizer, Inc. recently issued an announcement detailing the approval of its drug Ruxience (rituximab-pvvr), a biosimilar to the widely used monoclonal…
According to a press release from the Kentucky-based Apellis Pharmaceuticals, the Company has initiated a program to investigate the use of APL-9, an investigational drug, in gene therapies that involve…
According to a story from Raconteur, as many as 3.5 million people living in the UK are expected to affected by some type of rare disease at some point during…
According to a publication from Fierce Biotech, researchers from Boston Children's Hospital have created a new gene therapy technique they believe could provide benefits to patients with inherited forms of…
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Researchers at McGill University celebrated a “first” by creating a mouse model in a dish to demonstrate the effect that mutations in the SLC9A6 gene have on brain cells.…
In an exclusive "Good Morning America" interview this week, Dr. Priscilla Chan announced the launch of Rare As One, a new program from the Chan Zuckerberg Initiative where she is co-CEO…
According to a story from Pulmonary Arterial Hypertension News, data from an open label extension trial of the drug Orenitram as a treatment for pulmonary arterial hypertension has demonstrated that…
TGIF! Unless you're on vacation in which case, every day is *basically* Friday. This week, we're bringing four articles to read as you escape the summer heat. We have a…
The Immunization Action Coalition (IAC) Express Newsletter reports that 981 cases of measles have been reported so far this year, with 41 new cases added just last week. Rare disease kids…
According to a story from BioSpace, CTI BioPharma Corp. has recently concluded an End of Phase 2A meeting with representatives from the US Food and Drug Administration (FDA). This meeting…
The Sleeping Bear Dunes National Lakeshore Park, located in Michigan, has launched an innovated program to make their park more accessible to those living with a disability. They call it…
According to a story from BioPortfolio, the biopharmaceutical company CStone Pharmaceuticals has recently announced that China's National Medical Product Administration (NMPA) has issued approval for the company to conduct a…
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Many patients may not be aware of the vital role they play in medical discoveries, or that without their participation there would be no medical discoveries. CheckOrphan recently presented…
Last week, the New England Journal of Medicine published a report about a rare, disease-like infection that is a real cautionary tale. A 41-year-old British woman was diagnosed with acanthamoeba keratitis…
As recently reported in CheckOrphan, a new drug for sickle cell disease has quickly ascended through the FDA approval processes in an accelerated review. A New Sickle Cell Drug Shrouded…
Some promising news from across the pond! A new international consortium funded by the European Union will focus on speeding up the diagnosis and development of rare diseases and treatments.…
The Mental Health Rare Genetic Disease Network (MHRGDN) is a newly launched system, developed by the National Institute of Mental Health. The MHRGDN spans 15 research sites across the country…
Cyxone has just announced that the first person has been dosed in their Phase 1 clinical trial for Multiple Sclerosis (MS). This trial is investigating the effect of T20K. It…
According to a story from BioPortfolio, the company Novocure has recently announced that Medicare has released final local coverage determinations for its product Optune. Optune is used to treat patients…
No Cure, No Pay Emile Voest, an oncologist in the Netherlands, conceived the idea of a no cure, no pay model. After witnessing the exorbitant cost of drugs, some of…
Throughout my daughter Kelley’s 45-year life with a rare disease, there were numerous times that we needed to either call 911 or take her to the Emergency Department at a…
According to a publication from ET Healthworld, some 21 rare disease patients in India's northern state of Uttar Pradesh are still awaiting governmental financial assistance to which they are legally…
Rare diseases can leave patients feeling isolated and suffering in silence across the globe. A lot of work has been done to raise global awareness about common and devastating…
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