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…
According to a story from The Washington Post, two families affected by the rare disease spinal muscular atrophy have cause for celebration after UnitedHealthcare agreed to cover their treatment with…
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Genomic studies and advances in technology have increased researchers’ knowledge of several genetic syndromes that put people at risk for acute myeloid leukemia (AML). An article that appeared in…
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.…
Vertex Pharmaceuticals announced the submission of a New Drug Application (NDA) to the FDA for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen for treating Cystic Fibrosis (CF). This…
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…
Learning that you or a loved one has a rare genetic disease can be scary. Doctors often are not very well versed in these types of disorders and may require…
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…
15-year-old George Taylor reached out to NY Giants Running Back Saquon Barkley, telling the star NFL player how he's an inspiration to him for when he undergoes his chemotherapy for Ewing's sarcoma,…
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…
Cleveland Browns wide receiver Jarvis Landry has been affected personally by CF; he lost his high-school sweetheart Mya Zimmer in July of 2015 after her long battle with the rare…
According to a story from Science Daily, a recent study has found that osteosarcoma, a form of bone cancer, displays similar genetic characteristics when it appears in both humans and…
According to a story from Medical Daily, a recent study found that male Parkinson's disease patients that were sexually active tended to have less severe symptoms and slower disease progression…
According to a publication from the University of Wisconsin-Madison, analysis of a large outbreak of blastomycosis in Marathon County, Wisconsin in 2009 found that people of Asian descent were particularly…
According to a story from BioPortfolio, the biopharmaceutical company Genkyotex has recently announced that the US Food and Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application.…
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According to a report recently published in the Inter Press Service, since the advent of multidrug therapy in 1982, over sixteen million people have been cured of Hansen’s disease,…
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