Tay-Sachs disease and Sandhoff disease (also known as GM2 Gangliosidosis), and GM1 Gangliosidosis are neurodegenerative conditions. They are a result of a defective enzyme. This enzyme causes toxic gangliosides to accumulate within the…
Spinocerebellar Ataxia (SCA) refers to a group of neurodegenerative disorders. Ataxias are a component of the nervous system which control movement. SCA occurs when the ataxias in the brain or spinal…
The Myasthenia Gravis Foundation of America (MGFA) recently conducted a survey which found that most myasthenia gravis patients are not satisfied with their current treatment regime. These patients wanted a therapeutic…
Familial adenomatous polyposis (FAP) is a condition which causes extra tissue to form in various parts of the body such as the large intestine and gastrointestinal track. FAP affects about…
"People always say that 'cancer doesn't discriminate,' but the healthcare system often does," -Liz Margolies, LCSW, Executive Director, National LGBT Cancer Network. Unfortunately, the reality of this statement is that there…
According to a story from Market Screener, the drug developer Albireo Pharma, Inc., recently announced that its investigational product candidate A4250 has earned Orphan Drug designation from the European Commission.…
According to a story from the Translation Genomics Research Institute (TGen), the organization was recently awarded a grant of $10,000 from PayPal Gives. The funding will be allocated towards funding…
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…
Hope everyone's holiday season is off to a good start! As we gear up for Christmas and the New Year, we're bringing you four stories that stood out from this…
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…
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…
According to a story from pm360online.com, the pharmaceutical company Roche recently announced that its investigational therapy risdiplam has awarded PRIME designation from the European Medicines Agency (EMA). The drug has…
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…
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…
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…
Angelman Syndrome (AS) is a neurogenetic disorder. It's not fatal, but prevents patients' from living independently. It causes neurological impairment, seizures, and balance issues among other problems. It's caused by a…
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…
In August, Duke Cancer Center's Patient Support Program had a backlog of 150 wigs. That means 150 cancer patients who didn't have access to an item that, while simple, provides…
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…
Happy Wednesday! With Hanukah wrapped up, and Christmas just around the corner, things are getting pretty crazy. Take a second to relax and check out these four stories. First, we…
According to a story from BioSpace, the cancer drug company Gibson Oncology, LLC has recently announced that the US Food and Drug Administration (FDA) has awarded Rare Pediatric Disease designation…
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 Proactive Investors, the biotechnology company Conatus Pharmaceuticals recently reported that the company's drug Emricasan failed to achieve the primary endpoint in a Phase 2b clinical…
Sarcoidosis is an inflammatory condition which can affect any organ in people of any age. The prognosis ranges from benign to debilitating. Granulomas, or clumps of cells, build up in…
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