Graves' Disease Awareness Month is recognized in July of each year. As part of this year's awareness campaign, the nonprofit organization Prevent Blindness and Horizon Therapeutics collaborated to create the…
According to an article from BioTech 365, data from the Phase 3 INVICTUS trial and Phase 1 dose-escalation study, which evaluated QINLOCK as a treatment for gastrointestinal stromal tumors, was…
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…
This past week, Bristol Myers Squibb shared four-year data from the Phase 3 CheckMate-214 clinical trial. Ultimately, the trial sought to understand the safety, efficacy, and tolerability of Opdivo…
Last week, biotechnology company BioMarin announced that Dr. Lynda Polgreen, MD, MS would present at the American Society for Bone and Mineral Research (ASBMR) Annual 2020 Meeting. Her presentation centered…
According to a story from PR Newswire, the RNA-targeted gene therapy company Locanabio, Inc. has recently released findings from a preclinical evaluation of an RNA-targeted CRISPR Cas9 (RCas9) system as…
This past week, Cycle Pharma announced FDA approval of a once daily dose of NITYR for patients with hereditary tyrosinemia type 1 (HT-1). According to its prescribing information, NITYR…
Cynata Therapeutics Limited has recently announced the results from their preclinical trial of induced pluripotent stem cell (iPSC)-derived Cymerus™ mesenchymal stem cells (MSCs), according to BioSpace. They are testing these…
Recently, biopharmaceutical company Apellis Pharmaceuticals announced submission of a New Drug Application (NDA) and Marketing Authorization Application (MAA) for pegcetacoplan. If approved, these applications will allow for patients with…
Researchers have known, for a while, that a variety of gene mutations contribute to different subtypes of Charcot-Marie-Tooth disease. The specific condition is dependent on which gene is mutated. But…
A recent study published in the journal Blood Advances has documented that hematopoietic stem cell transplantation (HSCT) is both safe and effective for children with many different kinds of inherited nonmalignant conditions…
The American College of Obstetricians and Gynecologists (ACOG) has established new recommendations for pregnant women, including perinatal screening for depression as well as anxiety and mood disorders. A total of 40…
Safety of Sweeteners Sweeteners sound like the ideal solution to added sugar. No calories and none of the cons of refined sugar means you can enjoy it in excess right?…
We are learning more everyday about COVID-19. We know that for most people who contract the disease, recovery will be swift and they will return to normal rather quickly. However,…
Recently, clinical-stage biopharmaceutical company Aura Biosciences announced a Phase 2 clinical trial to determine the safety, efficacy, and tolerability of AU-011 for patients with choroidal melanoma. Currently, Aura Biosciences…
According to Ankylosing Spondylitis News, data from the Phase 3 PREVENT clinical trial highlights Cosentyx (secukinumab) as an effective treatment for patients with non-radiographic axial spondyloarthritis (nr-axSpA), as well as…
In a recent press release, biopharmaceutical company ChemoCentryx announced FDA approval for a New Drug Application (NDA) for avacopan, a treatment for ANCA-associated vasculitis. According to the FDA, a New…
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…
Jazz Pharmaceuticals has launched an educational program called Find the Right Fit, which aims to spread awareness and help patients with myelodysplastic syndromes (MDS) and secondary acute myeloid leukemia (sAML).…
In 2020, September 21-25 will be recognized as Global Immune Thrombocytopenia (ITP) Awareness Week. The week will aim to help spread awareness about ITP around the world both in the…
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Dr. Joan T. Merrill is a professor of medicine at the University of Oklahoma who presented on lupus at the 2020 Congress of Clinical Rheumatology - East. She spoke about…
Happy Friday! Sept. 24 is FH Awareness Day. Familial hypercholesterolemia is a gentic disorder characterized by the inability of the body to remove LDL -- sometimes called "bad cholesterol" --…
Several decades ago, gene therapy seemed to be on target to treat a disease by simply replacing a defective gene with a healthy one. A recent article republished in APNews…
Recently, pharmaceutical company Alimera Sciences announced that the first patient was enrolled in the NEW DAY clinical trial to test ILUVIEN as a potential treatment for patients with diabetic macular…
Last month, the annual ESC Congress, headed by the European Society of Cardiology, discussed innumerable treatments and advances in the field of cardiology. During the conference, biopharmaceutical company Kiniksa Pharmaceuticals…
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