Neuroscience News recently published an intriguing article suggesting that letrozole, a drug developed to treat breast cancer, has the potential to treat brain tumors. The discovery began with a question…
According to an article in Batten Disease News, enrollment is complete for a Phase 1/2 clinical trial. Within the trial, researchers will be evaluating Batten-1 for adolescents and adults with…
Osteosarcoma is genomically complex, which means that – sometimes – it can also be difficult to treat. Treatment and management, however, are crucial to ensuring positive patient outcomes. Doctors and…
Happy Friday! A tropical storm system will be traveling up through the mid-Atlantic this weekend, bringing wind and rain. Hunker down! This week, we have stories on Duke University being…
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…
Seven-year-old Rydder Ames of Hamilton, Ohio began having troubling symptoms five months ago. His mother, Krista Steven took him to the ER after a week-long headache followed by a…
According to a press release, the gene therapy company bluebird bio, inc. has recently announced that its gene therapy elivaldogene autotemcel (marketed as SKYSONA) has received Accelerated Approval status from…
FBXL4-related mitochondrial disease urgently requires new therapies. Unfortunately, this condition is often fatal by early ages (early to mid-childhood). Therefore, it is important to spur research into potential interventions or…
Currently, an estimated 187,000 children within the United Kingdom, and millions worldwide, have some sort of arm disability. This can be linked to damage or various conditions, such as cerebral…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to prevent, diagnose, or treat rare conditions. These are conditions affecting fewer than 200,000 Americans.…
According to an article from Cancer Network, the U.S. Food and Drug Administration (FDA) recently granted Orphan Drug designation to second-line GNS561 (ezurpimtrostrat) for cholangiocarcinoma (bile duct cancer). In…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
At 20 years old, Grace Hinchman was - for all intents and purposes - a happy and healthy young adult. Grace was studying Business at Loyola University Chicago and playing…
Seven-year-old Rydder Ames of Hamilton, Ohio began having symptoms five months ago. His mother, Krista Steven took him to the ER after a week-long headache followed by a loss of…
How does CAR T-cell therapy affect an enhanced anti-leukemia effect? Could higher doses influence toxicity or change patient outcomes? According to an article in the Pharmacy Times, researchers sought to…
Glioblastoma, a type of brain cancer, is notoriously difficult to treat. This is because the tumor is fast-moving, aggressive, invasive, and hard to remove with just surgery. However, an…
Dr. Sergio Baranzini of UC San Francisco recently led an international consortium of researchers in a new study of multiple sclerosis (MS). In a press release on Multiple Sclerosis…
Jim Kelly and his wife Maria still remember the day they received a call from the hospital telling them that according to test results, their daughter Adriana, now age…
A New Theory The Week reports that scientists at the Krembil Brain Institute in Toronto are working on a new theory about the origins of Alzheimer's disease. The Toronto…
Hi, my name is Becky. I am age 38 from the UK and am a proud wife to my wonderful husband Carl and mother to my three beautiful children:…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
It has been four years since Amaria Granger, now age 14, was diagnosed with a rare disease called Niemann-Pick disease type C (NPC). Since being diagnosed with NPC, Amaria…
Wunmi Bakare described her illness to Essence as a continuous tug-of-war. Balancing sickle cell disease with a nine to five job and trying to stay the course. Then there…
According to a story from neurology.duke.edu, Duke University has recently been recognized as a Center of Excellence for Dystonia treatment by the Tyler's Hope for a Dystonia Cure Foundation. This…
Did you know that September is National Sickle Cell Awareness Month? During this month, various stakeholders come together to share the stories of those affected by sickle cell disease,…
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