By Natalie Homan from In The Cloud Copy It’s estimated that about 3.4 million Americans have epilepsy, one of many conditions that can cause seizures. Seizures occur when there is…
A new study published in Blood has found different genetic variants of myelodysplastic syndromes (MDS). The most common of these is called SF3B1, which occurs in around 1 in every 5 patients. It…
May is recognized annually as Cystic Fibrosis (CF) Awareness Month. Each year, this rare disease community comes together in order to help spread awareness about cystic fibrosis among the general…
According to a story from Xconomy, the drug company PTC Therapeutics has come to an agreement that will allow it to acquire Censa Pharmaceuticals and its lead product candidate. This…
According to a recent announcement by AstraZeneca, the U.S. FDA granted its approval for Koselugo (selumetinib). This is the first drug to be approved for the treatment of children…
A new resource guide has been developed for X-linked hypophosphatemia (XLH) by Prime, a medical education firm. This project has been supported by Ultragenyx Pharmaceutical Inc. The guide provides resources…
The month of May is Huntington's Disease Awareness Month, in which a special effort is made to improve awareness about this devastating rare disease. This year, the Huntington's Disease Society…
According to a story from Buzzfeed News, the ongoing coronavirus/COVID-19 pandemic has put the brakes on critical rare disease research all over the globe, ranging from early, highly experimental studies…
Social distancing sucks for everyone. Even introverts (like myself) are becoming antsy. Similarly, the coronavirus is scary for everyone, even those less likely to be effected by serious illness. However,…
According to a story from the CureDuchenne Blog, CureDuchenne founder and CEO Debra Miller announced that the organization has recently entered into a new partnership with the biotechnology company Myosana…
By Danielle Bradshaw from In The Cloud Copy Sean and Josie Kelly, a New York native couple, have had a newborn son named Sawyer who was born with a rare…
According to BioSpace, healthcare technology company RDMD has raised $14 million in Series A financing, and developed a partnership with UCB. In addition to growing their platform to discuss…
According to a story from Barron's, the biotechnology company Alexion Pharmaceuticals has recently announced plans to test its drug Ultomiris as a potential treatment for COVID-19. As the scientific community…
According to a recent FDA press release, the agency has given its first approval for a drug developed specifically for adult patients diagnosed with upper tract, low-grade, urothelial cancer.…
According to a story from Live Science, the ongoing coronavirus/COVID-19 pandemic has upended daily life in a myriad of ways and can make leaving the house feel a little intimidating.…
According to a story from Biotech-365, the biopharmaceutical company Inventiva has been forced to change its plans to announce the latest updates for its phase IIb clinical trial. This clinical…
A new study is seeking to understand how the ongoing coronavirus/COVID-19 pandemic is impacting patients with a rare blood cancer called Waldenström's macroglobulinemia (WM). The research is to be conducted…
April is recognized as Fabry Disease Awareness Month by the Fabry community in the US and several other countries around the world. Advocacy groups such as the Fabry Support &…
According to a story from Biospace, the RNAi therapeutics company Alnylam Pharmaceuticals Inc. recently announced that its experimental product candidate vutrisiran has earned Fast Track designation from the US Food…
According to a story from PR Newswire, the National Organization for Rare Disorders (NORD) has just recently kicked off a COVID-19 Critical Relief Program. In alignment with its mission to…
According to a story from Porphyria News, Lucia Monaco, PhD, is the chair of the International Rare Diseases Research Consortium (IRDiRC). Despite the ongoing disruptions caused by the coronavirus/COVID-19 pandemic,…
According to an article in Science Daily, Professor David Nicewicz and a team of researchers at the UNC Department of Chemistry, discovered a method of creating tracers used in…
In a story written for The Washington Post, Jessica Slice writes about a frightful predicament for patients with chronic illnesses that cause disability. Jessica lives with dysautonomia and hypermobile Ehlers-Danlos…
A press release from the US Food and Drug Administration (FDA) recently announced the approval of the drug selumetinib (marketed as Koselugo) for the treatment of patients aged two years…
NICHD The NICHD is the The Eunice Kennedy Shriver National Institute of Child Health and Human Development, one of the National Institutes of Health (NIH). It is the center for…
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