According to a story from Specialty Pharmacy Times, data from a Phase II clinical trial has recently been released which suggests that the drug tagraxofsup is a useful treatment in…
According to a story from radio.wpsu.org, soprano singer Charity Sunshine Tillemann-Dick recently passed away at the young age of 35 because of pulmonary arterial hypertension, a rare condition of increased…
Diffuse Intrinsic Pontine Glioma Diffuse intrinsic pontine glioma (DIPG) is a rare form of childhood brain cancer that has a 100% mortality rate. Children diagnosed with DIPG only typically live…
Mantle cell lymphoma (MCL) and acute myeloid leukemia (AML) are two rare forms of blood cancer with a high unmet need. The majority of cases in these patient populations are…
Unknown Side Effects It's almost as if it's just a matter of time before a safety warning is issued for each new drug approved by the FDA stating an unexpected…
As I listened to her story I kept thinking, “You can’t make this stuff up.” Or at least no one would want to anyway. The first time I met Adeola…
According to a story from EurekAlert!, a team of scientists affiliated with the University of Cambridge have pioneered a new test that could herald a new development in the treatment…
According to a story from Chemical & Engineering News, the American Society of Gene & Cell Therapy annual meeting was more crowded than usual this year. In fact every event…
Happy Thursday! We hope everyone is enjoying this beautiful spring week! This week we're highlighting a story about a partner of ours that helps support creativity in kids with rare…
According to a story from BioPharma Dive, the advent of gene therapy development at Spark Therapeutics has been five years in the making. At this juncture, it has been nearly…
According to a story from BioSpace, the drug development company Dynacure recently released an announcement in which the company stated that its application for a Phase 1/2 clinical trial had…
According to a story from forum.facmedicine.com, Dr. Rahul Desikan has been dedicated to the study and research of neurological disease such as amyotrophic lateral sclerosis (ALS). Then, in a cruel…
According to a story from sciencemag.org, an experimental gene therapy for the rare genetic disorder X-linked myotubular myopathy is starting to produce significant improvements for patients. A devastating disease that…
According to a story from Science Daily, a team of scientists affiliated with UT Southwestern's Children's Medical Center Research Institute are hard at work pioneering an innovative new approach that…
A recent report that was published in the Swiss Medical Weekly outlines data gathered by a Swiss panel of experts on plasma cell myeloma. The report offers an update and…
According to a story from fox43.com, an experimental therapy for the genetic form of amyotrophic lateral sclerosis (ALS) has been termed "game changing." A study that will soon be presented…
Dutch Bros Coffee is hosting their 13th annual fundraiser for amyotrophic lateral sclerosis (ALS) this Friday, May 10th, 2019. It's called "Drink One for Dane" in honor of Dane Boersma,…
What Exactly is Mast Cell Disease According to The Mastocytosis Society (TMS), there are three forms of mast cell disease. All involve the malfunction or over-development of mast cells in…
According to a story from The Intercept, Ady Barkan's life is slowly slipping away as his amyotrophic lateral sclerosis continues to progress. Despite this, Ady has developed a reputation as…
Much of what we think counts as "treatment" in medical literature is informed by the amazing successes of high-tech drugs and therapies that have evolved in just the past few…
According to a story from Lambert-Eaton News, a recent large scale study using data from the Veterans Affairs has determined that the prevalence of Lambert-Eaton myasthenic syndrome (LEMS) is similar…
According to a story from People Magazine, parents Deborah and Antoine Vauclare were devastated when they found out that their son Léo was born with an incredibly rare genetic disorder called infantile…
According to a story from globenewswire.com, the biopharmaceutical company Innovation Pharmaceuticals recently released a report in which recent studies and pre-clinical research has suggested that the company's experimental drug Kevetrin…
Happy Thursday! This week we're highlight a variety of articles. First, we're starting with a strong call-to-action from contributor and advocate, Sharon. Next, we have an article about rare disease…
After 20 agonizing years, a mother and father have finally received answers regarding their daughter's health. It shouldn't take this long. The Diagnosis Their daughter was diagnosed with two very…
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