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…
We need to talk about adult rare disease patients. Adults who have a rare disease diagnosis are faced with a blaring issue-- I'm speaking for a large number of adults here,…
According to a story from the Andover Advertiser, mother June Beere has kicked off a campaign to open a cancer support center after her 42 year old daughter Gemma Kent…
Doctor Alan Pestronk has identified a new and very rare autoimmune disease. He is now trying to spread the word about its existence to other doctors in an effort to…
According to a story from BioPortfolio, the biotechnology company Tiziana Life Sciences plc recently announced encouraging results from an interim safety review of the company's Phase 2a clinical trial. This…
An Indian health policy initiative meant to provide funding to rare disease patients has struggled to gain approval from the country's administration, according to a publication from DNA India. First…
According to a publication from Karolinska Institutet, a medical research university near Stockholm, Sweden, new research led by the University suggests that regulatory T cells have an especially important role…
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Dr. David Porteous, a General Practitioner in Bristol, England recently reported to the Bristol Cable, a community-run newspaper, that he does not agree with the extensive list of treatments being…
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