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…
According to a story from PR Newswire, the genomics medical company Sangamo Therapeutics, Inc., recently announced that the first patient had been dosed in its Phase 1/2 clinical trial which…
According to a story from EurekAlert!, monitoring lung function is an important role of managing Duchenne muscular dystrophy and other diseases that can cause wasting of the respiratory muscles. Many…
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 freepressseries.co.uk, 45-year-old Stephen Millichamp was recently diagnosed with AL amyloidosis. The news was dire, and Stephen was told that the disease would ultimately be fatal.…
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…
The Heritage Florida Jewish News reported a story about one sister's journey to her Gaucher Disease diagnosis, thanks to her older sister's wedding prep. What is Gaucher Disease? Gaucher disease…
According to an article originally published by HDBuzz, a number of exciting advances have been made in the development of treatments that hope to treat thousands of potential Huntington's disease…
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 Financial Review, the US is not the only place in the world where lobbying is a central component of political activity. So far, lawmakers in…
Watch this and try NOT to get into the Christmas/holiday spirit! What makes this even better is that this annual Christmas lights display is put on to help raise money…
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…
According to a story from Dallas News, Shereese Hickson is a 39-year-old single mother. She has multiple sclerosis, and it seemed like her symptoms were starting to flare up again.…
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 Global Knock, nine year old Ashton Leeds has been living an active life again lately, but a year ago, things were not looking up for…
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 the National Post, 34-year-old mother Andrea Cardin, a photographer, was excited to welcome her newborn son James into the world on November 21st of this…
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…
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