A new treatment for Rett syndrome is officially being studied in humans in Canada, meaning Canadian patients may have a new, viable treatment option in the future. Called TSHA-102, this…
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…
Rhabdomyosarcoma is a rare form of cancer that affects children. It was recently studied at St. Jude Children's Research Hospital, where a team of medical professionals examined cells that persist…
Dr. Omar P. Haqqani, MD FACS is currently the Chief of Vascular and Endovascular Surgery at the Vascular Health Clinics. He has a keen interest in understanding and addressing vascular…
A recent article shared the story of a woman diagnosed with cystinuria whose life was saved after her younger brother donated one of his kidneys. Cystinuria Cystinuria is a rare…
It’s finally May – and you know what that means! That’s right, this month is Huntington’s disease (HD) Awareness Month. The theme for this month’s HD Awareness Month is #LetsTalkAboutHD.…
Ultragenyx Pharmaceutical Inc. has just announced the first dosing in their Phase 2/3 trial investigating setrusumab (UX143) as a treatment for osteogenesis imperfecta (OI) patients. This trial is called Orbit,…
Lennox-Gastaut syndrome (LGS) is a rare, severe form of epilepsy that is often resistant to anti-seizure medications. This means that patients still experience epileptic episodes despite treatment. Because of this…
Patient Worthy is delighted to announce that the National Society of Genetic Counselors (NSGC) has joined us as an advocacy partner! Genetic counselors play a critical role for individuals, families,…
Rare Pediatric Disease designation is granted by the FDA to drugs or biologics intended to treat life-threatening diseases affecting individuals aged 18 or younger. In this case, rare conditions are…
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…
As featured recently in the Cancer Therapy Advisor, a study has been conducted on patients diagnosed with metastatic renal cell carcinoma (mRCC), who also have elevated inflammation, and sarcopenia. The…
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…
Al Lupiano, an environmental scientist, recently spoke with Meg Baker of CBS2. Al told her that twenty years ago, he was diagnosed with a brain tumor. The memory and…
When Krista Webb was just two years old, doctors determined that she had some level of hearing loss. Her family was surprised about this; nobody else in her family, and…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
Prednisone is one of the more common corticosteroids prescribed to individuals with lupus to help reduce inflammation and decrease symptom burden. Corticosteroids are also prescribed for a number of other…
On April 28, 2022, the National Organization for Rare Disorders (NORD) and the Rare Disease Diversity Coalition hosted a webinar program titled "Walk in Our Shoes: The Experience of Rare…
It is no secret that there is an inequity when it comes to race and medical research. More often than not, research does not take all races and ethnicities into…
Don't forget to check out Part 1 of our interview with Katheron Intson, where we discussed her background, her research experience in GRIN1, and the reasons behind why she developed…
In a news release from May 3rd, 2022, rare disease and cancer therapeutic development company DepYmed Inc. shared that its lead PTP1B inhibitor, designed to treat Rett syndrome, was granted…
According to a recent article in Genetic Engineering and Biology News, Washington University and University of Hawaii researchers have uncovered a possible drug development candidate in soil bacterium called…
Acromegaly and gigantism are two disorders that share a main characteristic: abnormal growth. Because of this similarity, many people confuse one for the other. However, these are two distinct, separate…
The Fatty Liver Foundation (FLF) has just announced a new national survey which will document the experiences and needs of individuals living with non-alcoholic fatty liver disease (NAFLD) and nonalcoholic…
Necrotizing enterocolitis is a serious and potentially fatal intestinal disease which most often affects preterm infants. For many years, researchers have hypothesized that gut health – and in particular, the…
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