This is Jason’s story as told to Memorial Sloan Kettering’s “In The News”. Jason Mascolo at 34 years of age had been living with constant leg pain for years. However,…
In recent years, there have been advances in the treatment of pancreatic cancer. This is crucial, as the prognosis for this cancer is often poor, and the cancer itself is…
Researchers acknowledge that the primary cause of failure of stem cell transplants is disease relapse. This holds true specifically for allogeneic hematopoietic stem cell transplants where a cancer patient…
According to a story from Ophthalmology Times, the COVID-19 pandemic rapidly expanded the usage of telemedicine, including virtual, tele-ophthalmology appointments. It also highlighted the effectiveness of AI, and both of…
In the United States, Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare diseases or conditions. A rare condition is one affecting fewer…
According to a story from Rett Syndrome News, the pharmaceutical company Acadia Pharmaceuticals is seeking U.S. Food and Drug Administration (FDA) approval for its investigational therapy trofinetide. It has been…
Currently, it is possible to screen for Down syndrome during pregnancy. However, the screening measure used – ultrasound imagery – has less than 80% detection accuracy using common indicators,…
According to a recent article, a young boy who has been battling biliary atresia and was put on a liver transplant list has made miraculous progress. About Biliary Atresia Biliary…
In 2019, Rian and Mike Fee celebrated the birth of their son Anderson, who they call Andy. Since birth, Andy has always been such a light in the lives…
NBC2 has featured the story of pulmonary fibrosis patient John McDaniel since June. Now, McDaniel has received an incredible gift that will enable him to play his favorite sport…
1On July 27, 2022, global biopharmaceutical company GSK plc shared some exciting news via news release. The company’s therapy Benlysta (belimumab), designed for pediatric patients with lupus nephritis, is now…
On July 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar for July. As always, these recurring meetings help provide critical updates to the rare disease community…
A recent article highlighted the story of the world’s tallest man Sultan Kösen, in which it was revealed his growth is attributed to pituitary gigantism. Pituitary Gigantism Gigantism, also…
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…
A recent article is bringing light to the condition known as misophonia, a disorder which causes people to have severe responses to certain sounds. Misophonia Misophonia, which literally translates…
2157 words (source) vs. 423 words (mine) - 5% match In the Phase 2 LILAC clinical trial, researchers sought to evaluate the safety, efficacy, and tolerability of litifilimab (BIIB059) for…
At the end of July 2022, biotechnology company Biogen Inc. shared via news release some exciting updates in regards to its therapy tofersen, an investigational treatment for patients with…
When Caroline Cheung-Yiu's son Alex started to walk around age two, it was clear that he was struggling with his balance and coordination; but instead of improving, his issues started…
Recently a woman was admitted to Odisha hospital where she was successfully treated for her rare blood disorder aplastic anemia. Aplastic Anemia Aplastic anemia is a rare, but serious,…
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