PracticeUpdate recently published an article relating to a study with the primary goal of lightening the burden that many males experience regarding long-term prostate surveillance. Active surveillance has been preferred…
A biological marker ("biomarker") is a measurable trait or characteristic that can tell researchers something about your body or health. For example, body mass index (BMI) is a common biomarker…
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, Chiesi Global Rare Diseases and the biopharmaceutical company Protalix BioTherapeutics, Inc. have recently announced that they have launched an Expanded Access Program (EAP)…
On October 2, 2020, biotechnology company BioMarin Pharmaceutical ("BioMarin") announced that its investigational gene therapy candidate BMN 307 received the Fast Track designation from the FDA. The therapy is designed…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler June 10. My stem cell coordinator…
According to a story from BioSpace, the biopharmaceutical company Mereo BioPharma Group recently announced that its investigational therapy setrusumab has earned Rare Pediatric Disease designation from the US Food and…
Since 2012, the nonprofit organization Dysautonomia International has organized Dysautonomia Awareness Month. With the goal of spreading awareness of the condition in the medical community and among the general population,…
Seelos Therapeutics and Duke University have both signed a Sponsored Research Agreement, meaning they will work in tandem to evaluate and test SLS-004, a Parkinson's disease treatment. Their research aims…
Healio recently published interviews with members of UCLA’s DRC Health Training and Research Program conducted by Infectious Disease News. Only a few months ago, WHO had announced the end of…
October is Rett Syndrome Awareness Month, and even though the pandemic has changed the way that we celebrate it, there are plenty of ways to get involved! Rettsyndrome.org has organized…
According to a story from the AKU Society, the European Medicines Agency (EMA) has recently announced a positive opinion on extending the label indication for nitisinone (marketed as Orfadin®). The…
CTI BioPharma has just announced that they are submitting a NDA for Pacritinib as a new option for myelofibrosis patients who are facing severe thrombocytopenia. The thrombocytopenia is a result…
Recently, the Janssen Pharmaceutical Companies of Johnson & Johnson ("Janssen") announced the FDA approval of Simponi Aria (golimumab). The therapy is designed to treat pediatric patients (ages 2 or older)…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Let me begin by saying that…
Recently, Canada made strides towards better, more accessible treatments for patients with beta thalassemia when they approved REBLOZYL (luspatercept). Biopharmaceutical companies Bristol Myers Squibb Canada ("BMS Canada") and Acceleron Pharma…
The Value of a Service Dog September has been deemed National Service Dog Month. These animals are essential for many people living with chronic conditions, disabilities, and various rare diseases.…
A study reported in NewsWise evaluates retinoid-x receptor (RXR) agonists as a treatment for recurring and refractory multiple sclerosis (MS). This specific RXR agonist, bexarotene, was found to help remyelination,…
aHUS Tracking Atypical hemolytic uremic syndrome (aHUS) is a rare disease that causes blood clots to form within the small blood vessels in the kidneys. These clots lead to organ damage…
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 an article from BioSpace, the TargetCancer Foundation has began enrolling patients in their TRACK study. This study is intended to provide rare cancer patients with individualized treatment options…
Dr. Jasvinder Singh, a rheumatologist at Alabama’s University in Birmingham is the author of a study that was featured in Arthritis Care and Research. During a recent interview with MedPageToday,…
According to an article from BioSpace, Vivet Therapeutics and Pfizer have entered into a manufacturing agreement to develop Vivet's gene therapy, which treats Wilson disease. The terms of the agreement…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Dave Kinler and I have walked…
Damian Green from the University of Washington is the Principal Investigator for a new research project in multiple myeloma. This project is thanks to a research agreement between the Fred…
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