Is Gene Therapy The Key for Treating Sickle Cell Anemia?
According to a story from Nature, Dr. Elliot Vichinsky thinks that almost a third of his patients with sickle cell disease ultimately die from causes that could be prevented. Sickle…
According to a story from Nature, Dr. Elliot Vichinsky thinks that almost a third of his patients with sickle cell disease ultimately die from causes that could be prevented. Sickle…
Morgan's story Morgan Gleason is a 20-year-old living with Juvenile Dermatomyositis. It's a rare autoimmune condition causing weak muscles and rashes in addition to other symptoms. As a result of her…
Immune Thrombocytopenia (ITP) is diagnosed by low platelet counts in the blood. It also causes impaired platelet production. This put patients at risk for serious bleeding as well as spontaneous bruising.…
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 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 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 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…
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 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 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 NTD, a former NFL cheerleader successfully diagnosed her own rare disease, pectus excavatum, after her symptoms were dismissed by her doctors as nothing more than…
According to a story from philly.com, Markelle Fultz, who plays as a guard for the Philadelphia 76ers, was recently diagnosed with thoracic outlet syndrome. It has not been good news…
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…