Alpha-1 antitrypsin deficiency (A1AD) shares similarities with asthma, which leads to it often being misdiagnosed as asthma. This misdiagnosis leads to insufficient treatment. Toronto Western Hospital's Asthma and Airways…
Marinus Pharmaceuticals is a pharmaceutical company that works to create treatments for epilepsy and other neuropsychiatric disorders. They are in the process of conducting clinical trials to evaluate the safety…
According to a story from Buzzfeed News, social media giant Facebook has finally taken action against groups on the site that promote the use of black salve, an alternative treatment…
According to a story from Targeted Oncology, the results of a recent clinical trial have left drug developers Kite and Gilead with the confidence to proceed with regulatory approval from…
Karyopharm, a pharmaceutical company based in Newton, Massachusetts, has submitted a New Drug Application to the FDA for their drug XPOVIO, which is meant for the treatment of diffuse large…
Prevail Therapeutics recently submitted an Investigational New Drug application for their gene therapy called PR001. The FDA has recently notified them that this application is active, meaning that Prevail Therapeutics…
January has been designated as a time to bring awareness to alternating hemiplegia of childhood! Alternating hemiplegia of childhood is extremely rare, with about 1 in 1,000,000 reported cases; therefore…
Inventiva has recently completed Phase IIa of their clinical trial of odiparcil, a drug made for the treatment of mucopolysaccharidosis type VI (MPS VI). Iventiva is very excited about the…
Copyscape score: 4% Cancer is a disease that affects different people in different ways, and for some people, no existing treatments can effectively treat their cancer. GlaxoSmithKline has recently released…
Copyscape score: 2% Alnylam has recently completed the third phase of their clinical trial of lumasiran, which was created for the treatment of primary hyperoxaluria type 1 (PH1). While they…
As originally reported in MSU Today; in new experiments on mice, researchers looked into how the TS gene is related to autism. Tuberous sclerosis is a rare disorder known for…
As originally reported in the Hindu Times; getting healthcare for a rare disease is hard, in general. 95% of rare disease patients have no therapy options available. Thats 380 million…
In the world of rare diseases, progress can often feel like a slow and agonizing slog. In many cases, this is the unfortunate reality. However, a lot can change in…
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Pfizer issued a statement this week through its Media Relations that its drug VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy received positive opinion by the European Medicines Agency’s (EMA) Committee for…
As originally reported in Pulmonary Hypertension News, sleep is a natural cleanser for the body. During the nights, our brain imbeds our memories, removes metabolic waste, and heals itself and…
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