According to a press release from the German-American biotechnology company Immunic Therapeutics, the Company recently began a proof-of-concept trial for its investigational primary sclerosing cholangitis treatment, IMU-838. About Primary Sclerosing…
According to a story from Markets Insider, Eli Lilly and Company recently announced that the US Food and Drug Administration (FDA) has approved the company's drug ixekizumab (marketed as Taltz®)…
A new study conducted by researchers in Japan has uncovered a new potential way to diagnose Fabry disease. Fabry Disease Fabry disease is an illness which inhibits the patient from producing the…
Netflix debuted a documentary series this month called 'Diagnosis' -- based on a New York Times Magazine column -- which follows individuals with or caregiving for patients with a mysterious,…
According to a story from Biotech 365, the biotechnology company Stoke Therapeutics, Inc., recently announced that the very first patient has enrolled for the company's observational study of Dravet syndrome…
According to a press release from Eiger Biopharmaceuticals, the American Food and Drug Administration (FDA) recently granted the Company's experimental hepatitis delta virus drug (hepatitis D), peginterferon lambda, breakthrough therapy…
Aevolve, a medical innovation platform, announced last week that it will be making rare disease research a top priority in its blockchain-powered accelerator, which provides a pathway for alternative funding…
According to a story from Healio, a recent study has illustrated that the drug denosumab can improve the bone density of patients with primary biliary cholangitis (PBC) and autoimmune hepatitis…
It was announced late last week that Justice Ruth Bader Ginsburg had just completed three weeks of radiation treatment for tumors on her pancreas. Pancreatic cancer occurs in the pancreas, an organ…
There is a little boy in South Africa who is the only one in the country to be reported to have the ultra-rare neurodevelopmental disorder called alternating hemiplegia of…
According to a press release from Synlogic Therapeutics, the Company recently halted development of its investigational hyperammonemia treatment SYNB1020 after an unsuccessful phase 1/2 clinical study. About Hyperammonemia Hyperammonemia is…
Glioblastoma Glioblastoma is a rare type of brain cancer. Sadly, it is the most aggressive form. The median life expectancy for the disease is between 11 and 15 months and…
According to a story from abc.net.au, an unusual and poorly known rare disease has been making its presence known in the country of Australia. This disease is called mal de…
According to a story from Charcot-Marie-Tooth News, a recent study has revealed that patients with Charcot-Marie-Tooth disease (CMT) type 1A still display signs of muscle weakness even if they retain…
According to a story from Medgadget, a team of researchers associated with Yale University have discovered that the symptoms of Tourette's syndrome can be controlled through the use of functional…
Cure GM1 Foundation is hosting a family meeting filled with hope on Friday, September 20th! This gathering will be focused on community, clinical development and research updates, patient advocacy, and…
According to a story from Financial Buzz, the Steadman Philippon Research Institute (SPRI) recently received an award in research from the National Institutes of Health (NIH). The award is in…
According to a story from Mizzou News, Chris Lorson, who is the associate dean for Research and Graduate Studies at the school's College of Veterinary Medicine, recently earned four grants…
According to a publication from BioPortfolio, American biotechnology companies Akcea Therapeutics and Ionis Pharmaceuticals recently published final study results from their phase 3 clinical study of Waylivra (generic name volanesorsen)…
Families in England affected by Batten disease are struggling to access effective treatments in their home country. Nicole and Jessica Rich are two young girls who live with their families…
According to a story from Cancer Research UK, a combination treatment featuring the targeted cancer therapy venetoclax (marketed as Venclyxto) and the monoclonal antibody rituximab will soon be covered by…
TGIF! Something we believe is that the stories of rare disease patients are vitally important. This week, we're highlighting two articles written by patients, as well as a story about…
The day that changed who I am was a typical Saturday morning at the lake, mowing the lawn. May 4, 2013 was a day I just plain forgot. Well, I…
You take care of your skin to make it look flawless and healthy. Despite such efforts, your skin can catch disease without you being aware of it. One such disease…
According to a story from news-medical.net, a recent study is giving scientists new insights into the nature of Merkel cell carcinoma, a rare cancer that affects the skin. The study…
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