A recent study, published here in the journal Nature Medicine and used as the source for this article, has found that certain kinds of cells may be useful for predicting…
An experimental gene therapy program (called AXO-AAV-OPMD) being developed for the treatment of oculopharyngeal muscular dystrophy has been licensed with exclusive global rights to Axovant Sciences from Benitec Biopharma. There…
Zogenix, Inc has announced the top-line results from a Phase 3 study of the investigational drug ZX008 (low-dose fenfluramine hydrochloride) for the treatment of young patients with Dravet syndrome. The…
Recently in Charcot‐Marie‐Tooth news, progress has been made in identifying better testing to use as outcome measures for people living with Charcot-Marie-Tooth disease type 1A. A study called “Outcome measures…
According to a story from Deseret News, after she was diagnosed with multiple myeloma, Jenny Ahlstrom almost immediately had to make crucial decisions regarding her treatment: decisions that could mean…
Zynerba Pharmaceuticals, Inc. has announced their plans to begin a clinical trial of Cannabidiol in children and teenagers who have Fragile X syndrome. The study, called CONNECT-FX, is expected to…
According to a July 9 report by the Muscular Dystrophy Association (MDA), the MDA awarded a large grant to researchers studying Charcot-Marie-Tooth disease. The grant, totaling over $1,000,000, was awarded…
The company Pharnext SA, which is developing the investigational drug PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A, has agreed upon a paediatric investigation plan for the drug with…
According to a story from EurekAlert!, researchers have found that the drug Diamox, which is used to treat a variety of illnesses and conditions such as altitude sickness, glaucoma, epilepsy,…
Catabasis Pharmaceuticals Inc. has announced plans for a Phase 3 clinical trial called Polaris DMD. It will investigate the experimental drug edasalonexent for the treatment of patients with Duchenne muscular…
According to a story from ScienceDaily, a recent analysis has been conducted in order to investigate patient outcomes for children with the rare skin condition Stevens-Johnson syndrome, as well its…
The US Food and Drug Administration (FDA) has awarded Priority Review Designation to glasdegib, an experimental drug designed as a treatment for previously untreated acute myeloid leukaemia in combination with…
According to a story from Global News, direct-to-consumer genetic tests are becoming increasingly popular. These tests can be used to learn new info about your ancient ancestors, closer relatives, and,…
Results from research into an experimental therapy called SL-401 for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm have been shared at the Congress of the European Haematology…
Kalytera Therapeutics, Inc. has announced that a meeting with the Centre for Drug Evaluation and Research of the US FDA is planned for Tuesday, 31st July this year. The purpose of…
According to a story published by STAT, a daredevil neuroscientist at Stanford University is utilizing the relatively new concept of virtual reality to solve an unsuspecting problem. Not only is…
BrainStorm Cell Therapeutics Inc. has announced that it has secured its second U.S. manufacturing location for NurOwn®, an experimental drug currently under investigation for the treatment of amyotrophic lateral sclerosis…
According to a story from the Deccan Chronicle, researchers may have discovered a new treatment for Dupuytren's contracture that could offer substantial benefits over currently available treatment options. The scientists…
According to a story from medicalresearch.com, recent research has revealed that many pancreatic cancer tumors appear to have vulnerabilities to treatments that take advantage of genetic mutations that are present…
According to an article from the Detroit News, the most recent budget proposal from the Trump administration would call for cuts to Medicare to the tune of $236 billion over…
According to a story from pm360online.com, a new study of Prader-Willi syndrome will soon be under way thanks to a recent collaboration between the biopharmaceutical company Zafgen and the Foundation…
According to a story from Psychology Today, a study from 2017 titled Cognitive behavioral therapy is effective in misophonia: An open trial concluded that cognitive behavioral therapy could play a significant role in…
A recently published study found that, out of the people studied, young cancer survivors were more likely to develop endocrine diseases than people who had no history of cancer. The…
Researchers investigating the symptoms of testicular cancer have published their results in the British Journal of General Practise. Their study showed that testicular enlargement from a lump or swelling is…
According to Medical Xpress, Chris Jones from the Institute of Cancer Research in the UK has delved into the complexities of the rare childhood brain tumors called diffuse intrinsic pontine…
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