Learning about rare diseases is one of the first steps towards advocacy. If you or a loved one are affected by cold agglutinin disease (CAD), you may want to get…
The first patient has been dosed in a Phase 1 clinical trial, which is exploring Curis Inc.'s CA-4948 in conjunction with ibrutinib for hematologic malignancies. Examples of hematologic malignancies include…
11-year-old Oliver Shultz is recovering at home after two weeks of stress and fear. It began with an ache in his back, followed by a fever. Soon, the family was…
As shared in a press release by the American Academy of Neurology; the medical community had taken note of the rare brain pressure disorder idiopathic intracranial hypertension (IIH) when they…
According to BioSpace, Homology Medicines has released the first round of data from the clinical trials of HMI-203, a gene therapy being developed for the treatment of Hunter syndrome, also…
According to Charcot-Marie-Tooth News, researchers have discovered a new subtype of Charcot-Marie-Tooth disease (CMT) linked to the sorbitol dehydrogenase (SORD) gene. Read the full study findings published in Nature Genetics. SORD Gene…
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…
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