Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Rare disease patients often face a long, complicated, and frustrating journey to the right diagnosis and treatment. Ally Trainor, a 21-year-old student at George Mason, is no exception. She has…
When Camden Rau was born, his parents immediately knew that he was facing some sort of health problem. According to Camden's father, Steven, who spoke to Wink News, Camden's skull…
I love the holidays. The lights, decorations, family time... even the smells are better! People generally seem to be jollier, even though there are a few obvious stresses that come…
Ever since he was a young boy, JR Trout, who lives with spinal muscular atrophy (SMA), had a sense that there was something different about him. For years, he never…
Over the last decade, more information has been discovered about sudden unexpected death in epilepsy (SUDEP), although there is still much learning to be done. While researchers initially believed…
According to a story from blogs.biomedcentral.com, a research project called SCOPE H2020 focused on the impacts of chronic kidney disease screening of the elderly in the EU. The research included…
According to a story from spectrumnews1.com, three year old Israel Bombela has endured many health challenges in his short life so far. The youngster went through a bout of COVID-19…
According to a story from Newswise, the early results of a recent phase 1/2 clinical trial have revealed the potential of an experimental treatment that was being tested for blastic…
For those of us who suffer from depression, that mostly invisible mental illness that, at its worst, can render us incapable of getting out of bed and, at its best,…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a disease that affects the patient’s central nervous system, peripheral nervous system, and skeletal muscles or…
As reported in AP News, Bradford and Bryan Manning broke into a market in the fashion industry often overlooked: the blind. The glitz of fast fashion doesn’t work. So what are…
According to Fragile X News Today, a new registry recently launched within the United States to spur research into gene premutations linked to fragile X syndrome (FXS). Currently, the…
A recent study sought to evaluate the impact of family history on the risk of autoimmune diseases, particularly primary biliary cholangitis and autoimmune hepatitis. There hasn't been much research conducted…
The New England Journal of Medicine recently carried a report citing data from a Phase 3 clinical trial involving 307 people diagnosed with MSI-H-dMMR metastatic colorectal cancer. MSI-H (high levels…
The Yale Cancer Center has recently presented the findings of their study of the cost-effectiveness of caplacizumab at the 62nc ASH Annual Meeting and Exposition. Their conclusion, which is that…
Adam Settle, is an artist, football lover, and the youngest of eight. He’s also the subject of the new book, No Day Wasted: The Adam Settle Story, by Susan F. Stallings.…
A report from the Neurological Alliance calls for improvements in treatment and care for the 150,000 rare neurological disease patients in the UK, including spinal muscular atrophy (SMA) patients. It…
ASH 2020: CRISPR and Vertex’s Potential Cure for Sickle Cell Disease and More Glimmers of Hope Published: Dec. 7, 2020 BioSpace CRISPR Therapeutics and Vertex Pharmaceuticals presented new data on…
The United States is coping with an obesity epidemic. It is therefore critical to understand the distribution of body fat and its effect on treatment. A recent article in Healio…
On November 27, biotechnology company Vertex Pharmaceuticals Inc. ("Vertex") announced the approval of SYMKEVI with KALYDECO for the treatment of pediatric patients with cystic fibrosis (CF), says GuruFocus. The…
As reported in Warta Saya; for kids with cystic fibrosis (CF), a daily exercise needed to clear the lungs of the disease's characteristic build-up of mucus can feel long and…
by Lauren Taylor from In The Cloud Copy Pantothenate kinase-associated neurodegeneration or PKAN is a rare, inherited condition in which the affected child experiences progressive degeneration in certain parts of…
As of November 20, Zokinvy has been approved by the FDA for the treatment of Hutchinson-Gilford progeria syndrome (progeria) and a few other progeroid laminopathies in patients aged one year…
Bulgarian cystic fibrosis patient joins international campaign standing up for the rights of patients to access essential medicines Rositsa Malinova, a 22-year-old resident of Pazardzhik, Bulgaria, is taking on the…
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