Celgene Corporation has recently announced two exciting updates for the rare disease community! Update #1 The first big announcement from Celgene was that their supplemental New Drug Application (sNDA) for…
In November of 2018, England announced that cannabis could be prescribed for medicinal use by specialist physicians when no other treatments proved effective. It's prescription was purely to be decided…
Happy Wednesday Everyone! We're excited for the spring weather coming up! Today, we're highlighting an article on some good news for the rare community in canada, followed by an update…
Tay-Sachs disease is a rare, genetic and sadly fatal condition. It's a neurodegenerative disorder caused by an impaired production of the β-Hexosaminidase A enzyme (HEXA). Symptoms of this condition include listlessness, diminishing…
Medical devices include ECG electrodes, bandages, cardiac pacemakers, thermometers, and hemodialysis machines. A comparison of clinical trials shows that medical device trials are somewhat similar to pharmaceutical trials that evaluate drugs,…
Avraham Steinberg recently spoke at RARE2019, the 2nd International Congress on Advanced Treatments in Rare Diseases. It was held on March 4th and 5th in Vienna, Austria. His talk focused…
About Pulmonary Hypertension Pulmonary Hypertension (PH) is a form of high blood pressure which affects the arteries. Primary Pulmonary Hypertension refers to a type of the condition whose origin is unknown. Secondary…
Pemphigus Pemphigus is a rare autoimmune condition which causes the skin to blister. There are two main forms of the disease called pemphigus foliaceus (PF) and pemphigus vulgaris (PV). Pemphigus…
A few months ago, Patientworthy editor Kathy Devanny asked me some important questions about the assistance I receive from Patient Access Managers as a person living with the rare disease…
Multiple Sclerosis (MS) was long believed to be an illness that could only affect adults. In fact, the pediatric form of this disease was not discovered until just over 10…
Hypertriglyceridemia Hypertriglyceridemia is the term used to describe abnormally high numbers of triglycerides in the blood. People with Hypertriglyceridemia have an increased risk of heart disease, stroke, and heart attack.…
According to a story from nbcnewyork.com, scientific research on cells was always a challenging task. Trying to study a certain trait used to mean looking at a tissue sample, which…
New Research to be Presented In just a few days the American College of Cardiology will host their 68th Annual Scientific Session (ACC.19). It will be held in New Orleans,…
According to a story from Korea Biomedical Review, the Sanofi Genzyme Korea, a subsidiary of the pharmaceutical company Sanofi, recently issued an announcement stating that the South Korean Ministry of…
Ontario, Canada is in the process of working to revamp their healthcare system. One of their primary goals is to increase the role that patients and their families/caregivers play in…
According to a story from globenewswire.com, ProQR Therapeutics N.V. recently announced that it has begun dosing patients in its Phase 1/2 clinical trial. This trial is testing the company's investigational…
According to a story from BioSpace, the drug developer Horizon Pharma plc recently announced the release of topline data from a Phase 3 clinical trial of its experimental drug teprotumumab,…
According to a recent article in the Sunderland Vibe, the MRC Developmental Pathway Funding Scheme (MRC) was created to carry out the work of Professor Roz Anderson, who recently died of…
As much as we try to minimize human error in the healthcare field, it still happens. To decrease the chance of its occurrence we’ve created various policies, rules, and guidelines…
According to a story from news-medical.net, a results of a recent study could help accelerate the development of therapies for liver diseases based on RNA interference (RNAi). This study demonstrated…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is the collective term used to describe ulcerative colitis and Crohn's disease. These conditions cause diarrhea, rectal bleeding, fatigue, weight loss, and rectal bleeding. These…
According to a publication from McGill University and obtained via EurekAlert, a massive study of 1,280 individuals confirmed a strong, long-suspected connection between rapid eye movement (REM) sleep behaviour disorder…
Earlier this week, a press release from Alnylam Pharmaceuticals, Inc, announced the that the phase 3 study of their experimental acute hepatic porphyria drug Givosiran yielded positive results. According to the…
A publication from OncLive recently reported that the FDA has granted the experimental myelofibrosis drug Fedratinib a priority review designation, following Celgene Corporation's submission of a New Drug Application (NDA) to the…
A recent press release from the French biopharmaceutical company Inventiva announced that the Food and Drug Administration (FDA) had granted the company's experimental mucopolysaccharidosis VI (MPS VI) drug odiparcil rare…
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