Cassie Bebout is an Auburn University undergraduate student, on track to graduate this May. She chose the school for one primary reason- it is the home of Dr. Doug Martin…
Happy Wednesday Everyone! We hope everyone had a good St Patrick's Day. Today, we're highlighting a story from PW contributor Denise Crompton on grief. This is followed by an article…
Shifting Contracts Over the years the United States healthcare system has shifted more toward reimbursement models that are outcome-based instead of service-based. In essence, this means that instead of paying…
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).…
Progeria and Atherosclerosis Progeria, also known as Hutchinson-Gilford progeria syndrome (HGPS), is a rare disease affecting just 400 people across the world. It is caused by a mutated LMNA gene.…
According to a story from finanznachrichten.de, biotechnology companies are starting to set their sights on treating deadly diseases that are lacking effective treatment options, including some diseases that are considered…
Phase 1 Trial A Phase 1 trial by Gamida Cell is examining a new potential treatment option for patients with multiple myeloma and relapsed/refractory non-Hodgkin's lymphoma who have not responded…
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 story from Markets Insider, the biopharmaceutical company Orchard Therapeutics recently announced the presentation of proof-of-concept data in regards to their investigational drug candidate OTL-102. This experimental therapy…
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A recent announcement by the Ehlers-Danlos Society marks the beginning of the recruitment phase for an international study devoted to determining the root cause of hEDS. The gathering of this…
According to a story from BioPortfolio, Imbrium Therapeutics and Mundipharma EDO GmbH recently announced that the US Food and Drug Administration (FDA) has awarded Orphan Drug designation to their experimental…
According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…
According to a story from MedCity News, a patient with the rare blood disorder beta thalassemia will be the first patient dosed in a Phase I/II clinical trial involving a…
According to a story from Market Screener, the biopharmaceutical company Marinus Pharmaceuticals, Inc. recently announced its plans to initiate a Phase 3 study that is intended to test ganaxolone as…
What is Bronchopulmonary Dysplasia? Bronchopulmonary Dysplasia (BPD) is a diagnosis most commonly given to premature newborns. In children, BPD is also the most common cause of Pulmonary Hypertension (PH). Premature babies often develop…
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…
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…
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.…
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,…
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