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…
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 recent article in MedicalXpress, in 2004 Professor Sophie Lucas of the Louvain de Duve Institute began studying immunosuppressive cells that are known to block immune responses in…
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…
Sarepta Therapeutics has met with the Office of Tissues and Advanced Therapies of the FDA in order to discuss a Type C written response about a clinical trial for SRP-9001,…
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…
Josephine Bila was born with transfusion dependent beta thalassemia, a rare blood disorder. Recently, Josephine uploaded a video to Youtube in which she talks about her life as a patient,…
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