According to a story from MD Magazine, the team at Spatial Transcriptomics has utilized an innovative computational approach in order to map gene expression on the spinal cords of patients…
Bronchiectasis Bronchiectasis is a rare lung disease that currently has no cure and no effective treatment. It causes parts of the airways to become enlarged which results in lung damage.…
According to a story from Mirror Online, parents Gail and Matthew Rich were devastated when they heard the news that a critical treatment for Batten disease was rejected for coverage…
ACHM and XLRP Achromatopsia (ACHM) is a rare, inherited retinal disease which causes impaired cone photoreceptor function. Patients experience light sensitivity so extreme that it can cause blindness during the…
According to a story from news-medical.net, a pair of researchers affiliated with the University of Houston are planning to dedicate their time to improving detection and monitoring of lupus nephritis,…
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An article in Austin Gastroenterology recently asks this question. It also confirmed what we already know, that genetic testing is now available to the average person and in fact,…
Optiz C Syndrome Optiz C syndrome (OCS) is an ultra-rare condition whose symptoms are often mistaken for Bohring-Optiz syndrome, Kabuki syndrome, or Kleefstra syndrome. The disease affects patients physical and…
According to a story from Pulmonary Hypertension News, many patients with the rare disease sarcoidosis frequently experience lung complications. Included in these complications is pulmonary hypertension, a condition of elevated…
BOS Bronchiolitis obliterans syndrome (BOS) occurs in 50% of patients who endure a lung transplant. In essence, the patient's immune system attacks the bronchioles in the new lung, causing blockages.…
According to a story from kshb.com, Maureen Boesen was already prepared for the worst when she first took a DNA test in order to determine if she carried a BRCA…
Huntington's Disease & SCAs Huntington's disease (HD) and spinocerebellar ataxia (SCA) are both neurodegenerative diseases caused by the same type of error in the body's DNA. Essentially, the CAG or…
According to a story from Market Screener, the specialty pharmaceutical company Santhera Pharmaceuticals recently released an announcement in which the company revealed its intent to file for Conditional Marketing Authorization…
GO-ALIVE GO-ALIVE was a Phase 3 clinical trial examining the safety and the efficacy of Simponi Aria in ankylosing spondylitis (AS). Simponi was first approved by the FDA in 2009.…
According to a publication from the University of Edinburgh, a new study found a set of 37 genes that were highly expressed in breast cancer tumour-associated macrophages (or TAMs -…
According to a publication from EurekAlert, a recent study suggests a common, typically symptomless form of the herpes virus called cytomegalovirus may cause faster disease progression in cystic fibrosis (CF)…
According to a release from Sangamo Therapeutics and published by PR Newswire, experimental severe hemophilia A drug SB-525 proved to be well-tolerated and generally safe in a phase 1/2 clinical…
According to a press release from New Jersey-headquartered ADMA Biologics, the FDA has approved the company’s primary humoral immunodeficiency disease (PIDD) drug Asceniv for the American market. The company expects…
According to an article at HealthEuropa, including patients and their families as an important component of cystic fibrosis research is a requirement when developing future treatments for the disease. That's…
According to an article at ScienceDaily, a combination of immunotherapy and chemotherapy therapies caused pancreatic cancer tumors to shrink in the majority of patients in a phase 1b clinical trial.…
LEXINGTON, Mass. and AMSTERDAM, the Netherlands - uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug…
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An encouraging article recently published in Sickle Cell Anemia News announced that due to the urging of pharmaceutical companies and patient advocacy groups, the FDA has produced an updated draft…
According to a publication from Live Science, researchers from around the world are working on a way to identify cancer in the body more efficiently and reliably. One way scientists…
Females vs. Males with Fabry Fabry disease is an X-linked condition caused by a malfunctioning GLA gene. It was long believed to only affect men. Since women have two X…
Any fans of RuPaul Drag Race or the art of drag itself? Raise your manicured hand! Well, regardless of your answer - watch how the world of drag and rare…
GT Biopharma has just announced that their Phase 1 clinical trial for Mastocytosis, Myelodysplastic Syndrome (MDS), and Acute Myeloid Leukemia (AML) has been authorized to begin. Although this investigation had previously…
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