For small patient groups, creating momentum towards rare disease solutions often seems futile. To tackle such expensive and extensive endeavors, rare communities find strength and hope by putting their minds…
C. difficile is a bacterial infection that impacts the gastrointestinal system. It can lead to weight loss, diarrhea, dehydration, and even the loss of blood platelets. Megan Newman has had…
Passage Bio has recently announced that Orphan Drug Designation has been granted by the European Commission for PBKR03, a gene therapy for Krabbe Disease. This condition has yet to have…
Inbrain Neuroelectronics has announced that they have raised 16.8 million dollars which will support clinical testing of a new device for Parkinson's disease as well as epilepsy. This is a…
According to a story from Fabry Disease News, the month of April is recognized as Fabry Disease Awareness Month, a time dedicated to spreading awareness about Fabry disease in the…
According to a story from Pompe Disease News, April 15th will be recognized as the eighth annual International Pompe Day. This day is meant to spread awareness about Pompe disease…
The coronavirus pandemic’s effects have wormed their way into every aspect of life, and those of us who spend a lot of time engaging with the medical world know that…
The Usher 1F Collaborative has recently announced the opening of registration for its scientific conference titled "Therapeutic Strategies for Large Protein Coding Genes in Usher Syndrome." This event will be…
At the American Society of Hematology Annual Meeting in 2020 (ASH 2020), the latest info and data related to Gaucher disease, a form of lysosomal storage disease, was presented. It…
According to a story from Cushing's Disease News, the Cushing's Support and Research Foundation (CSRF) is organizing to support Cushing's Awareness Day, which takes place on April 8th. A day…
The month of April is recognized as Sjögren's Awareness Month, a time for spreading awareness about the autoimmune disease Sjögren's syndrome in the medical community and among the general public.…
According to a story from Markets Insider, the biopharmaceutical company CStone Pharmaceuticals has announced that China's National Medical Products Administration (NMPA) has approved avapritinib (marketed as Ayvakit) as a treatment…
The month of April is recognized as Sarcoidosis Awareness Month, a time for spreading awareness about sarcoidosis among the general public and the medical community. The Foundation for Sarcoidosis (FSR)…
Cancer Network recently reported that Jazz Pharmaceuticals’ Vyxeos was approved by the FDA to treat therapy-related pediatric patients one year or older who were newly diagnosed with acute myeloid…
Around 30% of all patients with relapsing-remitting multiple sclerosis (RRMS) experience some type of cognitive impairment or decline at the time of diagnosis. Around 50% of patients experience some of…
Primary Sclerosing Cholangitis Primary sclerosing cholangitis (PSC) is a rare condition which causes inflammation within the bile ducts of the liver. It can lead to cirrhosis of the liver or…
Financial Burdens in Rare Disease In 2019, 20% of all adults in the United States have reported that they had large medical bills that were unexpected. 18% currently had medical…
By May 25th, 2021 the FDA will announce their decision regarding approval of Opdivo used in combination with chemotherapy for gastrointestinal cancers. Back in December, Priority Review was granted to…
Aeglea Biotherapeutics has just announced a new collaboration with Immedica to bring pegzilarginase to other countries outside of the United States. They have created a license and supply agreement to…
Lyme disease, and other tick-borne illnesses, can cause intense and prolonged symptoms. In fact, some patients experience chronic symptoms for a number of years following the initial bite. However,…
Sometimes it can be difficult to find or afford certain medications designed for your condition. However, this may soon be changing for Canadians with spinal muscular atrophy (SMA). On March…
According to a story from express.co.uk, a treatment for phenylketonuria (PKU) is currently under consideration for coverage on the UK's National Health Service (NHS). The treatment is called Kuvan and…
According to a story from the National Hemophilia Foundation, the drug company uniQure has provided an update in regards to a case of hepatocellular carcinoma (HCC) that emerged in a…
Macey Brietenback, a resident of Bel Air, Maryland, is nineteen years old and has endured various operating procedures over 70 times. WJZ has been following Macey’s struggle with her…
According to a story from Medical Xpress, an uncommon fungal infection has been discovered in Alberta, Canada. This infection, called histoplasmosis, is transmitted through the fecal matter of bats and…
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