According to a story from Genome Web, a team of researchers from across the country have concluded an in-depth genetic analysis focused on the origins of Hirschsprung's disease, a rare…
According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…
Happy Wednesday! We're officially halfway through May if you can believe it! This week we're highlighting a piece about an intern using their rare experience to inform their work, followed…
According to a story from KUT News, Ava Shepperd and John Ben Shepperd, aged 14 and 18 respectively, always knew that they would eventually need to get kidney transplants. This…
The FDA has just recently approved CYRAMZA as a treatment for those with hepatocellular carcinoma (HCC), who have been treated with sorafenib, and who have ≥400 ng/mL of alpha-fetoprotein (AFP).…
Confusion, nausea, fever, anger, then back to normal: this was Lauren Vandiver’s life beginning in early childhood. Her parents, Phillip and Angela Vandiver, spent more than twenty years asking the…
H.R. 2408 H.R. 2408 is a new piece of federal legislation otherwise known as the "Ensuring Access to Quality Complex Rehabilitation Technology Act." Essentially, this act creates a new category…
David Rowland, age 77, is a cancer survivor and former veteran of the Air Force. He is also a tube feeder. People who use a feeding tube are considered disabled…
Scleroderma Scleroderma refers to a whole group of diseases, all rare, that cause the skin and connective tissues in the body to harden. This is a result of excess collagen…
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…
Researchers at the University of Colorado Anschutz Medical Campus have uncovered a never-before discovered immunodeficiency disease. How It Came About It started with a newborn boy who had a myriad…
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…
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A recent article in CBC News describes the heartbreak and struggle of a Canadian couple appealing to the Canadian government to fund medication for their younger son. Both their children, Andre…
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 Market Screener, the biopharmaceutical company Alexion Pharmaceuticals has recently announced some encouraging news regarding the status for ravulizumab (marketed as ULTOMIRIS) in the EU. The…
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…
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…
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