Want to learn more about recognizing symptoms and diagnosing patients? In a MedPage article, authors encourage readers to follow along on a case study to understand why a 58-year-old…
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,…
You've most likely heard of COVID-19. Although the numbers seem to constantly be growing, the current amount of diagnoses sits at just about 2.1 million people. In many articles, you'll…
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 an article in the Baker City Herald, a non-profit organization has launched a website designed to support and educate patients with bronchiectasis. NTM Info & Research advocates for patients…
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…
Uncertain times may be the understatement of the year so far. When the news of COVID-19 (coronavirus) caught our attention, there were a lot of people frozen in disbelief and…
According to a news release, researchers have found that cysteinase, a compound designed to treat a rare kidney stone disease, may also be effective in treating patients with pancreatic cancer.…
"An apple a day keeps the doctor away." Growing up, this sing-song refrain stood as nothing more than a reminder from my parents to eat my fruits and veggies. But it…
Seneca Biopharma, Germantown, Md., issued a press release announcing its recent meeting with the FDA to discuss elements of a final design and protocol for a Phase 3 trial investigating…
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 a story from Spectrum News, the Andrews family has been dealing with a rare disease called Niemann-Pick disease type C for years. Parents Pam and Chris knew early…
RARE on the Road: a Rare Disease Leadership Tour April 17th, 2020 Burlingame, CA The Rare Disease Leadership Tour is designed to give insights and education to rare disease advocates,…
According to an article in The Asahi Shimbun, Japanese scientists have developed a new vaccine to mitigate neuronal degeneration caused by Alzheimer's disease. The vaccine, delivered nasally, had promising results…
Ornithine transcarbamylase (OTC) deficiency is a disorder that can cause comas, seizures, and death, making it necessary for those affected to seek treatment. Arcturus Therapeutics aims to give those with…
According to a report by ABC News, Virtua Health, one of the largest healthcare providers in New Jersey, claimed to have a forty-year history of organ transplant completions. Nonetheless, a…
According to Oren Goldshtein, CEO of Vectorious Medical Technologies, remote patient monitoring is needed now more important than ever. Nobody knows how long the COVID-19 pandemic will last or…
A person with diabetes is not able to properly make or use insulin well. Advancements in science are now seriously looking at updating and perfecting ways to help people…
According to new research from the Journal of Infectious Diseases, a new protein epitope has been discovered while testing the immune response of people with Kawasaki Disease. Kawasaki Disease, also…
How will this affect my child? Will this diagnosis change the way my child can receive medical treatment? If an emergency arises, do I have the knowledge needed to…
A three cohort trial of STP705, a treatment for squamous cell carcinoma, has met its primary endpoint. This treatment is administered in a series of injections, offering an alternative to…
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…
According to a story from XTalks, pulling off a clinical trial testing a new drug in a scientifically rigorous manner is no simple feat. It requires the triangulation of a…
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