The United States FDA has just announced that they will be providing 6 new grants, which are worth 16 million dollars for rare disease research. These grants will last a…
According to a story from Market Watch, the biopharmaceutical company Alexion Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has approved a new formulation of…
On October 6, 2020, molecular engineering company Scribe Therapeutics announced an exciting partnership. Originally founded by molecular engineers and CRISPR inventors, Scribe Therapeutics seeks to develop in vivo genetic medicine to treat…
Fetal alcohol spectrum disorder (FASD) is a spectrum of disorders that can impact decision making and make individuals unable to understand the consequences of their actions. Because of this, it…
EB Research Partnership (EBRP) is based out of New York, United States. EB Research Foundation (EBRF) is an organization based out of Australia with the same goals. The two organizations have…
Currently, most treatments for scleroderma, a group of conditions causing the hardening of skin and connective tissue, are symptomatic. That is, they treat symptoms but not the underlying cause of…
On October 14th, 2020, the International Waldenström's Macroglobulinemia Foundation (IWMF) hosted a webinar featuring Dr. Irene Ghobrial of the Dana Farber Cancer Institute. The focus of her presentation was on…
In December 2019, the FDA approved Ervebo, the first-ever Ebola vaccine. However, despite preventative measures, there still was never any actually approved treatment options - until now. Recently, the FDA…
Recently, during the virtual 28th United European Gastroenterology Week Congress, the Janssen Pharmaceutical Companies of Johnson & Johnson announced 5-year results from a Phase 3 extension study on STELARA…
In early October, global biopharmaceutical company Bristol Myers Squibb announced the Canadian approval of ZEPOSIA (ozanimod) for patients with multiple sclerosis (MS). In particular, the drug is designed to…
Over this past week, the Harrington Discovery Institute announced five recipients of its introductory Harrington UK Rare Disease Scholar Award competition. The Institute, which is involved with Cleveland's University Hospitals,…
One of the biggest issues that many individuals fighting severe COVID-19 face is an overactive immune response. In effort to protect itself, the body creates dangerous levels of inflammation. Then,…
Dr. Justin Hopkin's son Garrett was born happy and healthy, but when he was around four to six months old, his parents began to notice some problems. Garrett was having…
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…
About one month ago, the HealthWell Foundation announced the beginning of the Adrenal Insufficiency Fund. Designed to assist patients with adrenal insufficiency, the fund will grant financial assistance to…
A team of scientists have created a nanoparticle tool which allows scientists across the country to study COVID-19 without any risk of infection. This particle mimics how the virus infects…
J.G. Jones is a renowned comic book artist, illustrating covers of beloved superhero novels like Batman and Robin, Doc Savage, and Frankenstein. He has probably worked for all of the…
Happy Friday! October is going by quickly, but if you need something else to help pass the time, why not give our podcast a listen? The two newest episodes of,…
According to a story from GlobeNewswire, the biopharmaceutical company Immunomedics, Inc., has recently announced that its medication sacituzumab govitecan-hziy (marketed as Trodelvy) has recently earned Orphan Drug designation from the…
Ionis Pharmaceuticals has recently released the data from their clinical trial of IONIS-ENAC-2.5Rx, a treatment for cystic fibrosis. According to Newswire, the study showed very positive results, with healthy volunteers…
According to a story from GlobeNewswire, the gene therapy company Axovant Gene Therapies Ltd. recently announced that its gene therapy AXO-AAV-GM2 has been given Rare Pediatric Disease designation by the…
Researchers have just uncovered 30 new genes that may cause Charcot-Marie-Tooth Disease (CMT). These genes were uncovered using computer analyses of biological data, a process called bioinformatics. This study was published…
A recent study published in BMC Rheumatology has found that a combination therapy could have a benefit for individuals diagnosed with diabetes and ankylosing spondylitis (AS), psoriatic arthritis (PsA), or rheumatoid arthritis…
While many pharmaceutical companies submit approval and marketing applications, not all are successful. According to Fierce Biotech, a subsidiary of Questex, Santhera Pharmaceuticals ("Santhera"), is one of the most recent…
In recent years, there have been huge progressions in the field of gene therapy. At its core, gene therapy addresses genetic disorders by using genes to directly treat or…
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