According to a story from news-medical.net, a recent study has reshaped the scientific understanding of the origins of mitochondrial disease. For decades any mutation that affected the mitochondrial DNA and…
According to a story from Market Screener, the biopharmaceutical company Biohaven Pharmaceutical Holding Company Ltd. recently announced that the first patient has been enrolled in the company's Phase 3 clinical…
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 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…
A study published in the Orphanet Journal of Rare Diseases monitored the long-term progression of 16 patients that had Niemann-Pick Disease type B. The study served to gather important observational data…
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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 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…
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…
A recent publication from the Massachusetts Institute of Technology announced promising results from research into a novel new type of therapy called RNA interference (RNAi). RNAi could potentially be used…
A recent article published in online CANCER NETWORK reports findings that indicate children who have survived Hodgkin lymphoma (HL) may develop solid tumors in later years. These findings will have…
According to a story from globenewswire.com, the drug developer The Cell Factory and the University of Padova in Italy have been conducting research in order to demonstrate the effectiveness of…
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…
According to a story from dubainewstoday.blogspot.com, the data from a Phase I clinical trial suggests that an experimental drug candidate from the pharmaceutical company Boehringer Ingelheim could substantially improve the…
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…
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,…
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.…
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,…
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