According to a story from BNN Bloomberg, data from three studies suggest that Zolgensma, a gene therapy for spinal muscular atrophy developed by Novartis, could be useful to all patients…
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…
The first ever drug in the United States for treating transthyretin amyloid cardiomyopathy (ATTR-CM) has finally been approved by the FDA. It's amazing news for this patient community. But, you…
The 9th Annual Great Plains Rare Diseases Summit This event is geared towards the rare disease patient community that is situated in the great plains region. The summit will be…
Cedric Rutland, a pulmonologist at Pacific Pulmonary Medical Group has shared his experience, research, and thoughts on cognitive bias in an effort to spread awareness for the issue, and hopefully,…
Transthyretin-Mediated Amyloidosis Cardiomyopathy The FDA has just approved the very first treatment for hereditary or wild-type transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM). ATTR-CM is a rare disease that occurs when amyloids, an…
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…
Rare Disease Advocacy Day May 8th, 2019 Are you a patient from Alabama? Well this is your opportunity to speak with lawmakers directly about your concerns and what you think…
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…
The 2019 Congress of Clinical Rheumatology held May 2nd through the 5th in Destin, Florida featured a talk by Yusuf Yazici, the Director of the Behçet Syndrome Center at the…
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,…
Rare Disease Support Group May 6th, 2019 Featuring guest speaker Lemzel Johnson Living with a rare disease can be a daunting and isolating experience. Why not seek out other people…
In February, Li Kegiang, Premier of the State Council, announced that the Chinese government was going to take steps to improve rare disease patients access to medicine. As part of…
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…
RARE on the Road RARE on the Road is a Rare Disease Leadership Tour that will be making several stops throughout the US. The goal of this event is to…
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A recent posting by the Leukemia and Lymphoma Society references the Beat AML Master Trial. The trial evaluates targeted therapies to treat acute myeloid leukemia (AML) and was launched…
Uveitis Uveitis is a rare condition in which the uvea of the eye becomes inflamed. The uvea is responsible for providing the majority of the blood to the retina and…
An Unexpected Emergency Kristin Hardy and her sister Margaret are no strangers to rare disease. Kristin has primary progressive multiple sclerosis (MS) and Margaret has relapsing-remitting multiple sclerosis. Margaret also…
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…
Sarcoidosis Sarcoidosis is a rare inflammatory condition that causes granulomas to buildup within the organs. These can cause permanent organ damage. While the condition can affect various organs, and presents…
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…
According to a story from Yahoo, the family of Wyatt Beiermann, a seven year old boy with lethal mitochondrial disease, is doing their best to savor the limited time that…
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