TG Therapeutics has been developing ublituximab as a treatment for chronic lymphocytic leukemia (CLL) when used in combination with umbralisib, marketed as UKONIQ. The biopharmaceutical company has recently completed and…
Jaxson Corcoran was born with tuberous sclerosis complex (TSC), a rare genetic disorder that is characterized by benign tumors forming throughout the body. Treating this condition has become a family…
Tim Revell has run the Ascension Seton Austin Marathon a whopping 15 times, with his 16th run planned for this Sunday. The father of two runs the race for his…
Gene therapy is a recent development in the industry, and it is one that has brought hope to many people who live with a genetic condition without a viable treatment…
A study published in Physician's Weekly posed the question: is removing milk from the diets of children with eosinophilic esophagitis a preferable treatment option? A team of researchers, led by…
According to BioSpace, a consulting firm called Fact.MR has conducted a study into the market for acute hepatic porphyria (APH). The point of this study was to understand the future…
The warm weather is finally here (for most of us), which means it's almost time for fun, outdoor activities! We're not the only ones who are excited to spend time…
True was diagnosed with congenital amegakaryocytic thrombocytopenia (CAMT) when she was just six months old. Now she desperately needs a bone marrow transplant to survive. Her mother, Anessa Haden, is…
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A decade ago, Rob Long's dreams were centered solely on football. He was looking forward to the NFL draft after a successful college career at Syracuse University, but unfortunately, a…
Scholarships are being provided to rare disease patients, thanks to the EveryLife Foundation for Rare Diseases. The scholarships are each worth $5,000 and are intended for those seeking an education…
According to Medical Xpress, a team of researchers from The Wenner-Gren Institute at Stockholm University and Karolinska Institutet, Karolinska University Hospital has conducted a study that investigates neuronal death in…
Gene editing, CRISPR specifically, has become much more well-known and heavily used in the recent past. Advances in this field have allowed medical professionals to treat conditions that had seen…
According to Healio, an open-label study of oral octreotide has reported that the treatment is able to maintain its response in acromegaly patients without additional safety signals. This data is…
Lyme disease is a vector-borne disease spread through ticks. It is most common in the Northeast, North-Central, and mid-Atlantic portions of the country, but is notably absent in the Southeast.…
Sawyer Burch is a four-year-old boy from the Nashville, Tennessee area who was born with trifunctional protein deficiency, a rare disorder that takes the body's ability to utilize certain types…
A recent study published in Frontiers in Immunology investigates the immune response to bone infections, such as Staphylococcus aureus, that cause osteomyelitis. Their research stands to give medical professionals a better…
A study published in Physician's Weekly investigated the visual outcomes, clinical features, and treatment of individuals with occlusive retinal vasculitis (ORV), a condition that is characterized by inflammation in the vessels of…
One of six-year-old Jackson Garwood's favorite things to do is listen to his father sing. Some of his favorite numbers include 'Talk Tonight' by Oasis and Aqualung's 'Brighter Than The…
Three people in the state of Indiana live with Niemann-Pick disease type C (NPC), and Audrey Mischler is one of them according to WTHI-TV 10. She was diagnosed very recently,…
The National Institute of Health (NIH) has created a new approach to diagnosing and treating methylmalonic acidemia (MMA), a rare genetic disorder that stops the body from metabolizing fats and…
Timothy and Andrew Revell are two brothers born with Duchenne muscular dystrophy (DMD), a rare neuromuscular disorder that sees the progressive wasting and weakness of the muscles. This condition has…
Arimoclomol is a treatment that is in development for ALS, Niemann-Pick disease type C (NPC), and inclusion body myositis (IBM). A recent Phase 2/3 trial has ended in failure, unable…
The FDA has recently provided 89bio with guidance for their upcoming clinical trial of BIO89-100, a treatment for nonalcoholic steatohepatitis (NASH). The trial will advance as planned, beginning in the…
Steven Elvidge has been left confused, frustrated, and debilitated for the past three years due to a mystery illness. Various symptoms have left him unable to live independently, forcing him…
Rare disease patients throughout India have expressed their disappointment and anger towards the country's new policy centered on rare conditions. One of their main concerns is that the policy changes…
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