A new treatment for acromegaly has recently become available for patients. Called MYCAPSSA, this delayed-release capsule is intended for long term maintenance of acromegaly. Considering that injections are the typical…
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…
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…
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?…
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…
The Global Genes RARE Patient Advocacy Summit September 21-23, 2020 This conference will be a convergence of a variety of rare disease stakeholders, such as rare disease patients, world renowned…
Glut1 Virtual Community Gatherings: Spanish Language Group Q&A with Adult Patients & Families September 19th, 2020 The Glut1 Deficiency Foundation is not letting the coronavirus/COVID-19 pandemic stop them from continuing…
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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…
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…
Tyrosinemia is a metabolic disorder which prevents the body from processing the amino acid tyrosine. Without treatment, the disorder can be lethal. There are three different types of tyrosinemia with…
Chronic kidney disease patients often develop aortic stenosis, which requires a transcatheter aortic valve replacement (TAVR) procedure to treat. The question arises: will a TAVR procedure worsen the renal function…
According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…
Dane Tidwell lost his leg to osteomyelitis when he was just 18 years old. He spoke about the confusion and loneliness that came with being an amputee, and how his…
According to a story from BioSpace, the biopharmaceutical company Aridis Pharmaceuticals, Inc., has recently announced that it has struck an agreement with the US Food and Drug Administration (FDA) in…
It didn't take long for Jeff and Katis D'Angelo to realize that something was up with JJ, their newborn son. The biggest indicator that something was off was that the…
An article that appeared recently in Psychology Today asks whether dementia is responsible for falls. The answer brings to mind the many medical and environmental factors that affect the elderly…
September is recognized as Charcot-Marie-Tooth disease (CMT) Awareness Month. Although this illness is one of the most widespread inherited genetic disorders, there is still a significant need to spread awareness…
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