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,…
An unsettling fact: every three minutes, someone in the United States is diagnosed with some form of blood cancer. September is Blood Cancer Awareness Month to help raise awareness…
Happy Friday! The days are getting shorter and the first cooling winds of autumn have started to blow. This week, we have stories on Charcot-Marie-Tooth Disease Awareness Month, a special…
Before you read on, make sure you've checked out Part 1 of our interview, where we discussed what eosinophilic esophagitis is, its symptoms and diagnostic criteria, and why Dr. Dellon chose to…
The Orphan Drug Act of 1983 was designed to facilitate the development of orphan drugs, or drugs designed for patients with rare or life-threatening conditions. Now, the FDA grants…
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…
Before you read on, please make sure to check out Part 1 of our interview, where we discuss what a desmoid tumor is, and share Amy's story of living with a desmoid…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
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