Professor Timothy Yu developed the custom drug milasen named in honor of Mila, an 8-year-old girl with Batten disease. The drug is the first drug specifically designed for one…
Lonnie's mother, Stephanie Pesterfield of Colorado Springs, still finds it difficult to talk about her son Lonnie’s near-death experience. Yet as published recently in People, Stephanie and her son want…
Growing up, Jack Witzig saw how his father inspired others. His dad Don was a cross country and track coach at two different high schools. He motivated his students to…
Family gatherings can be fun, boisterous, and sometimes full of conflict. During one such event, Donnie Adams found himself intervening to break up a fight between two people. Although he…
When Chantel and Jason Funk learned that their son Bowin has adrenoleukodystrophy (ALD) in 2019, they thought that the questions about the future, and how to manage the condition,…
Marco and India Biviano felt like something bad was happening to their young son Luca. His stomach was distended and painful. Luca frequently developed chest infections that required round after…
Leukemia is cancer that begins in the bone marrow and involves abnormal white blood cells (WCB) from lymphatic tissues and bone marrow. Scientists have new theories as to why certain…
Prothena Corporation which is based in Dublin, Ireland recently announced, via Business Wire and Biospace results of the VITAL Phase 3 clinical trial showing a significant survival benefit…
Findings from the Max Delbrück Center and the Charité Universitätsmedizin Berlin have been published in the European Respiratory Journal stating that a triple combination therapy can improve CF symptoms…
If your child was sick, you’d do everything that you could to save them. Hayley Moore is no different. When her daughter Klarissa was born, Moore felt such a…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
According to a report in the morningstar.com, the FDA has approved AstraZeneca/Sanofi shot that protects infants and toddlers against RSV. The 2022/2023 winter saw an RSV increase, especially among…
According to a story from European Pharmaceutical Review, the European Commission has recently approved a label extension of ORKAMBI (lumacaftor/ivacaftor), a drug used to treat cystic fibrosis. Under the extension,…
Congenital muscular dystrophy (CMD) affected individuals, Kelly Berger and Avery Roberts are making their voices heard. Together they hope to tackle misconceptions surrounding disability and give a loud voice…
Currently, there are an estimated 10,000 known rare diseases (and counting). As we learn more about our genetic makeup, and how genetic alterations can cause disease, we continue to…
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…
It’s no secret that our gut health plays a formative role in our overall health. Over the past decade, more and more studies have examined how the gut microbiome impacts…
An estimated 15% of people with sarcoidosis, a rare condition which causes granulomas to form throughout the body, develop neurosarcoidosis. This form, which is characterized by central nervous system (CNS)…
In June 2023, the U.S. Food and Drug Administration (FDA) approved the use of VYVGART Hytrulo for people living with generalized myasthenia gravis (gMG), a chronic autoimmune neuromuscular disease…
The Peter Pan novel which J.M. Barrie wrote in 1911, based on his 1904 play, includes a curious concept about the hideout trees Peter uses for his lost boys' housing.…
Dr. Brooke Molina and co-authors at Pittsburgh University have reassuring news for parents of children with ADHD. The team conducted an in-depth study, recently published in JAMA, that followed…
According to a story from news-medical.net, a recent study found that elevated body-mass index (BMI) was associated with worse outcomes for people living with acute lymphoblastic leukemia, with this study…
According to a July 11 article from Maaisha Osman, commercial-stage biotechnology company ADC Therapeutics has voluntarily halted enrollment for the Phase 2 LOTIS-9 study evaluating Zynlonta (loncastuximab tesirine-lpyl) and…
On the field at the University of Tennessee, defensive back (DB) Trevon Flowers was known for his intense gameplay, strong athletic ability, and defensive nature. He appeared in 46…
English-American guitarist, singer, and songwriter Peter Frampton is best known for his hit album “Frampton Comes Alive!” and his upbeat, immersive performances. But he is also known for his efforts…
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