Aibe Shreve is only four years old, and he has already overcome so many obstacles in his short life. The little boy from Ohio Valley was born with two holes…
Vance Robinson was diagnosed with inclusion body myositis over ten years ago, and since then he has worked to raise money and awareness. Pre-pandemic, Vance would throw the first pitch…
Researchers have not always understood why some people develop Addison's disease, a rare autoimmune condition affecting the adrenal glands, over others. Now, a new study conducted by Swedish and Norwegian…
Pfizer and Imcyse have entered into a collaboration and licensing agreement for a rheumatoid arthritis (RA) program that utilizes Imcyse's Imotope technology. The two companies have worked together in the…
Michael Freeland spent most of his life trying his best not to think about his incredibly rare disorder. Not only was it incredibly painful, but the symptoms were debilitating, and…
In the past, patients with short bowel syndrome (SBS) were treated with teduglutide, a daily injection. However, injections can be painful, difficult to administer, and unfortunately not as targeted.…
Developed by United Therapeutics Corporation ("United") and DEKA Research and Development Corporation ("DEKA"), the Remunity Pump is now commercially available for patients with pulmonary arterial hypertension (PAH). On February…
In a recent press release, pharmaceutical company Moleculin Biotech, Inc. ("Moleculin") shared a recent advance in the sphere of medical and health research. Agencja Badań Medycznych, a Polish agency which contributes…
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…
February 15th is recognized annually as International Angelman Day, a day dedicated around the world to spreading awareness among the general public and in the medical field about Angelman syndrome,…
During clinical trials, researchers work to advance clinical knowledge, build an understanding of certain diseases or conditions, and learn more about a new drug, surgical device, or behavioral modification technology. However,…
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…
The United Kingdom has taken a big step in the right direction for rare disease patients, as they have recently created the UK Rare Diseases Framework. This framework is intended…
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…
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…
According to ForexTV, commercial gene therapy company Spark Therapeutics ("Spark") recently announced preliminary data from a Phase 1/2 clinical trial evaluating SPK-8016, an investigational gene therapy, for patients with hemophilia…
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…
The American Society of Clinical Oncology will soon hold their Genitourinary Cancers Symposium (ASCO GU) from February 11 through 13, 2021. The symposium, held virtually during COVID-19, will feature…
In early February, global healthcare company Insightec shared that the company received FDA approval for its new study. Within the study, Insightec will evaluate Focused Ultrasound as a potential…
After in-licensing Icatibant, a Bradykinin B2 receptor agonist, from Slayback Pharma, global pharmaceutical and biosimilar leader Sandoz is launching a generic version in the United States. According to a…
According to Oncology Nursing News, a new study performed by researchers at the Baylor College of Medicine used germline testing to identify genetic precursors to pediatric rhabdomyosarcoma. The study identified…
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