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…
According to a story from ArcaMax, history was recently made when a 15 year old teen with a severe "superbug" case of Mycobacterium abscessus infection was successfully treating using specially modified…
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…
According to a story from Patch, Mother's Day 2019 was an exceptionally special one for Gwen Finlayson who received a gift from her son Brandon that will give her a…
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A recent article in the neuroscience section of the Medical Express reports the results of a study conducted by researchers in the Charcot-Marie-Tooth/Hereditary Neuropathy Center at internationally acclaimed Cedars-Sinai.…
Three stories of courage are highlighted in Orlando Family Magazine’s annual tribute to the medical community. The first example relates to a mother, Eva, for her courage in opting…
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It has been less than two years since Cytokinetics’ previous ALS muscle drug, tirasemtiv, failed to meet its endpoint in a Phase III trial to treat ALS. (That was after…
According to a story from Yahoo News, the Patented Medicine Prices Review Board (PMPRB), a small agency of the Canadian government that employs less than 100 people in total, is…
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…
According to a story from Biopharma Dive, an investigational therapy for Huntington's disease that is in development could be the first step in the implementation of the first-ever disease modifying…
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…
According to a story from m.dailyhunt.in, Vinayak Sreedhar, an Indian student with Duchenne muscular dystrophy, passed away in the midst of taking his exams. Vinayak was a driven and highly…
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 Multiple Sclerosis News Today, 2019 is the tenth year that the Multiple Sclerosis Association of America (MSAA) has conducted its Art Showcases initiative. The program…
According to a story from The Irish Times, a couple's application for permanent residency in Australia has been rejected twice now following an appeal. Anthony and Christine Hyde, along with…
According to a story from globenewswire.com, the drug developer Zynerba Pharmaceuticals recently announced that the company has been awarded Fast Track Designation from the US Food and Drug Administration (FDA).…
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 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…
According to a story from Medical Xpress, a recent study has suggested that the drug eculizumab could substantially reduce the risk of disease relapse in patients with the rare disease…
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…
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…
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