Rett Syndrome Rett syndrome is a rare and progressive neurodevelopmental disorder caused by mutations to the MECP2 gene. While it affects all ethnic and racial groups, it is found almost…
According to a story from prnewswire.com, the biopharmaceutical company Dompé recently announced that the company has received the 2019 Industry Innovation Award from the National Organization of Rare Disorders (NORD).…
According to a story from BioSpace, the biotechnology company Minoryx Therapeutics recently announced that the company has received official approval from the Spanish Agency of Medicines and Medical Devices (AEMPS)…
According to a publication from Multiple Sclerosis News Today, German biotechnology company Immunic Therapeutics has successfully enrolled the first participant for its phase 2 clinical trial for experimental relapsing-remitting multiple…
According to a recent article in the Globe, the Danish biotech company Saniona AB has initiated a Phase 2a clinical trial of Tesomet for the treatment of hypothalamic obesity, a rare…
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 press release put out last month by American pharmaceutical company Adverum Biotechnologies announced some of the long-term preclinical safety data collected about their experimental drug ADVM-022. The drug hopes…
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