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…
Tarsius Pharmaceuticals has just announced positive results for their recent trial for uveitic glaucoma. This trial was called GADOT 20/20 and it was investigating a therapy called TRSo1. Ocular Diseases…
Last week, clinical stage biopharmaceutical company Neurotrope announced that the first patient was dosed in a Phase 2 clinical trial to test the safety, efficacy, and tolerability of bryostatin-1…
Grifols, a pharmaceutical company based out of Barcelona, Spain, has announced that their potential treatment for the novel coronavirus has entered a phase 3 trial, according to BioSpace. It is…
According to a recent article in BioSpace, young children and infants who have been diagnosed with primary hyperoxaluria type 1 (PH1) are often faced with the use of gastrostomy…
Patient Worthy has recently begun a partnership with the Glanzmann's Research Foundation, a nonprofit patient organization dedicated to spreading awareness about Glanzmann's thrombasthenia and finding a cure. We spoke with…
According to a story from GlobeNewswire, the biopharmaceutical company X4 Pharmaceuticals, Inc. has just announced that the US Food and Drug Administration (FDA) has granted its investigational product candidate mavorixafor…
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