Prader-Willi syndrome (PWS) is a neurodevelopmental disorder which causes intellectual disability, a deficiency of growth hormones, compulsive behavior, a high risk of obesity, and hyperphagia, a symptom which can be…
According to a scientific study published in the Orphanet Journal of Rare Diseases, a team of researchers were able to identify the mutations responsible for choroideremia in five families of Polish descent.…
According to a story from HudsonAlpha Institute for Biotechnology, a team of researchers affiliated with the organization have identified a genetic mutation that is linked a rare pediatric form of…
According to a story from EurekAlert!, a collaboration of Sage Bionetworks, The Children's Tumor Foundation (CTF), and the Neurofibromatosis Therapeutic Acceleration Program (NTAP) have announced the initiation of the very…
According to a story from EurekAlert!, a team of researchers used a combination of microarray technology and blood tests in order to distinguish distinctive molecular signatures in a group of…
A new Phase 2 Parkinson's disease trial is now enrolling 42 patients who have lived with the condition four years or more. Ultimately, the trial aims to evaluate a new…
A University of Florida research team has announced its results of a gene replacement therapy in the treatment of Barth syndrome, which delivered promising results in its initial phase of testing.…
According to a story from Oncology Nurse Advisor, a recent study found that patients with immune thrombocytopenia do not carry the same understanding as their doctors do when it comes…
According to a story from BioSpace, Incyte Corporation and Eli Lilly and Company recently announced that the US Food and Drug Administration (FDA) has given the drug baricitinib Fast Track…
According to a story from news-medical.net, a team of researchers has found that patients who are more intellectually active can slow the neurodegeneration that appears in Huntington's disease. Such as…
Neurodegenerative disorders affect millions of people across the world. They include Amyotrophic Lateral Sclerosis, Parkinson's disease, Huntington's disease, and Alzheimer's disease, among others. For most neurodegenerative diagnoses, there are no effective treatments, no…
According to a story from Charcot-Marie-Tooth News, a group of scientists have developed a new scale that can be used to predict disease severity in infants that are diagnosed with…
For a long time, we've known that, at least in North America, men develop cancer at higher rates than women. However, there is still some mystery concerning the impact of…
According to a story from Debra of America, a new Phase 1 clinical trial being operated by Stanford University is currently recruiting participants. This trial will be a test of…
According to a story from Charcot-Marie-Tooth news, researchers have found that models of mutations affecting the mitofusin 2 protein (MFN2), which is the protein that is affected Charcot-Marie-Tooth disease type…
According to a story from pm360online.com, the drug developer Zynerba Pharmaceuticals, Inc., recently announced the release of data from an open label, 12 month study of the company's experimental product…
According to a story from bioengineer.org, a team of researchers from around the world have collaborated and successfully discovered a critical mechanism that leads to lung inflammation following exposure to…
Angelman syndrome impedes the brain's ability to distinguish background noise from whats important. This is a result of a reduced tonic inhibition, caused by a loss of function of the…
According to a story from the Centers for Disease Control and Prevention, the National ALS Registry is playing a vital role in learning more about amyotrophic lateral sclerosis, a devastating…
According to a story from Newswise, scientists have previously discovered that there are two pathways that colorectal polyps may utilize as they transform into colorectal cancer. These are dubbed the…
Hereditary Angioedemia (HAE) results when the blood is missing the C1-inhibitor protein. It causes unpredictable and sudden attacks of swelling on various parts of the body. It can affect practically…
To be quite frank, Lyme disease is an under-researched condition. If you look at the data concerning how many clinical trials are developed each year for rare conditions, Lyme disease…
One Chinese scientist has completed a research project that has shocked many in the medical community. Two twin baby girls born in November of 2018 where the recipients of his…
According to a story from pm360online.com, the gene therapy company Fibrocell Science, Inc., recently received an investment of $900,000 from the Epidermolysis Bullosa Medical Research Foundation (EBMRF) and the EB…
According to a story from curetoday.com, a recent Phase 2 trial has demonstrated that bortezomib (marketed as Velcade) has the potential to be an effective therapy for patients with mantle…
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