The 2023 Special Olympics BC Winter Games took place from February 2-4, 2023. Sporting events at Winter Games include 5-pin bowling, curling, figure skating, alpine or cross-country skiing, and more!…
Dr. Valeria Ricotti of the Institute for Child Health- Great Ormond Street UK, spoke with BBC News on the subject of AI analyzing body movement. Dr. Ricotti and other researchers…
On January 26, 2023, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
The month of February is recognized as Turner Syndrome Awareness Month, a time to spread awareness about this rare disease among the medical field and the general public. The Turner…
This is Part 2 of a two part story. Check out Part 1 here. The loss of his child through Edwards Syndrome helped Dr. Harsha Rajasimha understand the suffering and…
Known as “Slydah,” Chicago White Sox relief pitcher (RP) Liam Hendriks has done amazing things on the field. In September/October 2021, Hendriks was named the American League Reliever of…
The number of identified rare diseases continues to climb every year, with the number recently reaching the 11,000+ mark. Some of them have only a handful of identified cases, and…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
ABC News recently carried an article describing an anticipated surge in the latter part of 2020 of acute flaccid myelitis. Thankfully, it did not occur. The disease is caused by…
For many people living with mantle cell lymphoma (MCL), the standards-of-care include Bruton's tyrosine kinase (BTK) inhibitors. But there is a subset of patients who do not respond well,…
PART I The baby died almost immediately after birth. Edwards Syndrome is a rare congenital disease. Only about ten percent of newborn babies survive past their first year. Babies…
When Arica Svoboda thinks of her two children, Hayes and Hendrix, she can’t help but fill up with love. She tells Patient Worthy: “My boys are special. Their smiles and…
Have you ever heard of Marfan syndrome? This rare inherited disorder affects connective tissue (fibers that support and anchor organs and other bodily structures) throughout the body. Marfan syndrome most…
For many people, a rare disease diagnosis can feel like life is over. For Angel, growing up with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition characterized by extremely high…
Friends and family refer to 41-year-old Teddy Higgins as kind, selfless, and loving. Given all of his support for others, his friends and family are now working to support Teddy…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In a recent news release from biotechnology company Cartesian Therapeutics, the company shared that the first patient was dosed in a Phase 2b study evaluating Descartes-08 for generalized myasthenia…
When Rylae-Ann was one year old, her parents had to hold her in an upright position at night in order for her to breathe. Rylae-Ann had a rare genetic disease,…
Treatment for Alagille syndrome, a rare genetic disorder that affects multiple organ systems, are typically symptomatic. Many children born with this disorder eventually require a liver transplant; without this, Alagille…
According to a story from St. George News, when painter Linda Toombs was diagnosed with systemic scleroderma, a rare autoimmune disease, doctors told her that she wouldn't see her children…
An opinion expressed by Sheila Frame, President of Americas at Amryt Pharma, was published recently in the Pittsburgh Post-Gazette. Ms. Frame, who serves on the board of the Rare…
Neuromyelitis optica spectrum disorder (NMOSD) can have long-term impacts for those affected. Current research suggests that up to 69% of patients may have severe unilateral or bilateral vision loss…
Enzyme replacement therapy (ERT) has been explored as a potential therapeutic option for mucopolysaccharidosis type III A (MPS III-A or Sanfilippo syndrome type A). However, these solutions have not…
What would you do if you were told that your child had a rare disease? How about if your child had an ultra-rare disease, which affects fewer than 20 in…
The other day I read a post in a dystonia support group that is similar to something I see and hear very often. I’m sure you have as well, and…
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