A biopharmaceutical company based in California called Retrophin recently announced that the first patient has been administered treatment in their phase III study in to test RE-024 (fosmetpantotenate) as a…
Making scientific discoveries must be a lot like trying to map a coastline in the dark. There’s a lot of bumping into things and getting your feet wet in the…
Multiple Sclerosis is a disease of the brain and spinal cord. In patients with MS, their immune system attacks the protective myelin sheath that usually covers nerve fibers. These nerve…
You've got to be dedicated to train for a marathon. When Brit Joe England signed on to raise pounds for the Alkaptonuria (AKU) Society, he was optimistic about finishing the…
A study by the Sotiria Chest Diseases Hospital in Athens Greece revealed a surprising new link between asthma and bronchiectasis. Visit bronchiecstasisnewstoday.com to read the full article and study results.…
The U.S. FDA just announced that it has accepted Genentech’s supplemental New Drug Application (sNDA) and has granted Priority Review for Zelboraf (vemurafenib) in patients with Erdheim-Chester disease from the…
Even though the diseases that Patient Worthy covers are considered “rare,” collectively, the rare disease community affects millions and millions of people worldwide. Despite this, there is a huge socioeconomic…
August signifies the beginning of the end of the summer season and the start of school again, but it is also a month of spreading awareness. Specifically, gastroparesis awareness. Gastroparesis is…
Chronic granulomatous disease (CGD) is an inherited primary immunodeficiency disease associated with granulomas, which are clumps of immune cells that form at the site of infection or inflammation. As a…
Most people take breathing for granted — until the ability to breathe is impaired. People with idiopathic pulmonary fibrosis (IPF) know this all too well. Without the ability to readily…
Mitobridge, Inc., a pharmaceutical company that focuses on improving mitochondrial function, just announced the achievement of a vital milestone in the first-in-human clinical trial of PPAR-delta modulator, MA-0211. This study…
You’re more likely to recognize his voice rather than his face. If you love segments like “Food News with Froggy” or “Phone Taps” from the Elvis Duran and the Morning…
Waldenstrom macroglobulinemia (WM) is a devastating form of non-Hodgkins lymphoma. Specifically, this cancer starts in the B cells of the body (B lymphocytes). Waldenstrom's cancer cells are capable of making…
So I don’t think it will surprise anyone to learn that people living with mental illnesses like schizophrenia or bipolar disorder have to put up with a huge ration of…
The Children’s Oncology Group (COG) and the National Cancer Institute (NCI) and have begun collaborating on a new project, to be known as the Pediatric Molecular Analysis for Therapy Choice…
Neuroblastoma, though rare, is the most common type of cancer in children who are five years old or younger. This cancer targets the immature nerve cells in the nerve tissue…
Tissue engineering has a relatively easy concept behind it. First, a patient’s stem cells are grown in the laboratory. Then, they are added to a scaffold material. And ta da!…
Jon Milne is on a quest to race across the seven seas to help raise awareness of rare disease! Jon has always been a keen sportsman, aged 30 he had…
Global Genes is hosting its 6th Annual RARE Patient Advocacy Summit for the rare disease community! Here's all you need to know about it, and why you won't want to miss it:…
Sometimes, all it takes to succeed is knowing that someone is depending on you. For example, numerous studies have shown that there is better quality of life for elderly people…
It started out harmlessly enough—a Greatest Loser- type competition between co-workers. Everyone who wanted to participate put in $10, weigh-ins would be weekly, and the person who lost the most…
Don’t eat that! Eat this instead. Some diets recommend low fat. Some endorse high protein. Some forbid carbohydrates. Some praise those same carbohydrates. Some limit fiber. It’s so confusing figuring…
A new therapy option for hereditary angioedema (HAE) has recently received FDA approval! Marketed as Haegarda by biopharmaceutical company, CSL Behring, this subcutaneous C1 Esterase Inhibitor is now usable in…
BioMarin just announced interim results from a continuing Phase 1/2 study for hemophilia A patients, testing a gene therapy known as BMN 270. Hemophilia A is a rare bleeding disease…
The other day while I was shadowing a neurologist, I encountered a multiple sclerosis (MS) patient. The patient's MS was stable and he was doing quite well on his medications.…
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