Raghav is a two-year-old boy born with an ultra-rare disease caused by a mutated GPX4 gene. He and eight other children are the only ones in the world who have…
A study recently published in The FASEB Journal and led by Dr. Chiharu Nishiyama from Tokyo University of Science has demonstrated that a compound found in blueberries could serve as a…
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…
As reported in Biospace, a collaboration between biopharmaceutical company AstraZeneca and BenevolentAI has for the first time used AI to identify a molecule that plays a key role chronic kidney…
Ocular Therapeutix has announced that they will attend and present at the Angiogenesis, Exudations, and Degeneration 2021 Meeting, which is being held virtually due to the pandemic. They intend to…
A survey created by Eurodis, a European rare disease advocacy alliance, has shown that those living with a rare disease have a worse healthcare experience than people with a chronic…
Biotech company Ovid has released disappointing results from a trial of OV101, a potential treatment for Angelman's syndrome. The Phase 3 trial demonstrated that there was no significant difference in…
Recent research has discovered a new, potential treatment option that could provide an early intervention for Beckwith-Wiedmann syndrome and Russell-Silver syndrome. Published in Cell Reports, this study could provide the…
Alterity Therapeutics has recently announced the the Michael J. Fox Foundation will be funding a dose optimization trial for ATH434. According to BioSpace, this grant is the second from the…
Scientists are researching Alzheimer’s disease (AD) from all angles and gradually closing in on the cause of the disease. According to an article in Neuroscience News, a discovery by researchers…
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…
Patient groups can optimize patient registries, cross-sectional studies, and/or longitudinal natural history studies to maximize collaborations with sponsors of orphan drugs and gene therapies. Written by Harsha Rajasimha, MS, PhD…
The International Waldenstrom's Macroglobulinemia Foundation (IWMF) recently hosted a webinar presentation on February 9, 2021. This webinar, titled "Why Clinical Trials Matter and How to Find the Right One for…
Ionis and the University of California, San Diego (UCSD) have formed a team to investigate antisense oligonucleotide technology as a treatment for multiple myeloma. Their drug, ION251, has shown positive…
AVROBIO has released data from the ongoing Phase 2 FAB-GT trial, which is investigating AVR-RD-01 as a treatment for Fabry disease. This ex vivo lentiviral gene therapy was shown to…
According to a story from GlobeNewswire, the biopharmaceutical company Soleno Therapeutics, Inc. recently hosted a Key Opinion Leader (KOL) webinar, which was focused primarily on the latest updates to its…
Hundreds of mutations have been connected to autism spectrum disorder (ASD), and a new study finds further evidence that suggests a possible cause of this spectrum of conditions Neuroscience News…
An accumulation of alpha-synuclein is a major characteristic of Parkinson's disease, one that is often used to diagnose the condition. Current methods see medical professionals measuring the rate at which…
Zymeworks Inc. has recently began enrollment for an expansion cohort of the Phase 1 trial of ZW49. ZW49 is an antibody-drug conjugate (ADC) that targets HER2 positive cancer cells. This…
Improving cognition and identifying abnormal molecular pathways in Alzheimer’s disease has been challenging. Current studies of Alzheimer’s disease have focused on reducing such occurrences as amyloid plaque, neuroinflammation, and neurofibrillary…
Microcephaly is caused by a depletion in neural progenitor cells (NPCs). This depletion is caused by a mutation in the centrosome genes, and it occurs while the brain is still…
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…
According to a press release from Sanofi, platform and poster presentations focused on the company's investigational product candidate avalglucosidase alfa are slated to be made public at the 17th annual…
A study has recently highlighted the fact that patients who have type 2 diabetes are more susceptible to chronic kidney disease (CKD), and as a result, cardiac issues. This study…
The very first patient has just been dosed in Pfizer's Phase 3 clinical trial for Duchenne muscular dystrophy (DMD). The trial is called CIFFREO. This patient received the therapy at a…
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