According to an article published in the Orphanet Journal of Rare Diseases, a workshop held on June 23, 2019 in Orlando, FL focused on a difficult subject: improving detection and diagnosis for…
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…
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…
In an interview with Healio, Dr. Jennifer Miller says that there are signs of hope for improved outcomes in patients living with Prader-Willi syndrome, a rare genetic disorder. Dr. Miller…
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 story from The Charlotte Weekly, local resident Kevin Tobin recently ran 400 laps around his house---the equivalent of a marathon---to help raise money for Dan Donoher, a…
In a post on the Metachromatic Leukodystrophy (MLD) Foundation's website, the organization discusses the possibilities of using gene therapy as a treatment for this rare genetic disorder. The page discusses…
According to a story from Hematology Advisor, the drug luspatercept (marketed as Reblozyl) recently demonstrated its ability to reduce transfusion burden in patients with beta thalassemia that depend on regular…
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.…
On Wednesday April 22nd, 2020, the World Orphan Drug Congress held a one hour webinar that was focused on how the rare disease community could respond to the ongoing coronavirus/COVID-19…
Según una historia de Malvern Daily Record, la compañía biofarmacéutica Millendo Therapeutics, Inc. publicó recientemente datos de su ensayo clínico de fase 2b. Este estudio estaba probando la terapia experimental…
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…
The ME Association has recently published its latest weekly "research roundup" for the week of April 17th. This includes the latest research related to myalgic encephalomyelitis (ME), also known as…
For Mallory Holloway, precautions to avoid lung infections have been a part of her daily life for a long time. This is because she lives with cystic fibrosis, a rare…
According to a story from Yahoo News, there's no denying that the coronavirus/COVID-19 pandemic is an unprecedented experience for millions of people around the world. However, it can be especially…
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 koco.com, the community of Ada, Oklahoma, recently held a birthday celebration for a local affected by Alexander disease. Hunter Goodwin recently turned 18 years old,…
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…
The ongoing coronavirus/COVID-19 pandemic has caused widespread uncertainty across the globe, and a lot of us have questions about what to do or what to expect. The rare disease community…
Según una historia de CureDuchenne, la compañía de biotecnología Dyne Therapeutics anunció recientemente que CureDuchenne Ventures ha realizado una inversión de capital que respaldará el desarrollo de todas las nuevas…
April is Sjögren's Syndrome Awareness Month. This is held annually in an effort to spread awareness about this syndrome, and has been held since 1998. Unfortunately, this disease receives little…
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,…
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