Sancilio recently completed enrolling children with sickle cell anemia/disease in a Phase 2 clinical trial to evaluate a new therapy called Altemia (docosahexaenoic acid). The SCOT trial, as it will…
Global Genes is hosting its annual Tribute to Champions of Hope event for the rare disease community. Here's all you need to know about it: What: A blue-tie cocktail hour and dinner event…
Hema Biologics have recently released something even cooler and more inventive than just their traditional hemophilia treatments. They are way on top of their game and know exactly how to…
What should you know about Tourette syndrome? How can you explain it to your child? These are probably just a few questions you may have if your child has been…
Happy Friday! This week, we have a story that might break your heart about a woman who battled cystic fibrosis. But we also have a story that might inspire you about a…
Much like the United States’ National Organization for Rare Disorders (NORD), E-Rare in the European Union was launched over a decade ago to bring together organizations that fund research for…
According to Dr. Steven Carson, of all the autoimmune disorders that are both chronic and joint-related, Sjogren's syndrome remains the hardest to manage. Standards of care designed to guide doctors…
The United States FDA just granted Orphan Drug Designation to Napo Pharmaceuticals for Mytesi (crofelemer) in the treatment of short bowel syndrome (SBS). SBS is a rare disorder in which…
Orphan drugs, for those of you who don’t know, are those designated especially for rare diseases. They respond to a public health need, but not necessarily an economic need by…
In recent news in the oncology community, the FDA has approved orphan drug designation to a new drug, SurVaxM, for glioblastoma. Glioblastoma is a rare type of brain cancer that…
Sa-Nakht was an ancient Egyptian pharaoh who lived circa 2700 B.C. While there are many tall tales surrounding his reign, one that is especially tall, literally, and not figuratively, is…
If you remember reading our story on 12-year-old Markie DeVoe and his choroideremia, we have an important update for you. If you haven't read the story yet, you can find…
A new study pinpoints faulty genes as another potential cause behind hemiplegic cerebral palsy. Until now, there was an insufficient amount of information to verify that genetics was part of…
Theravance Biopharma recently proudly announced the results of a successful Phase 2b trial for their gastroparesis treatment, Velusetrag. The results showed that this treatment safely and effectively improved symptoms and normalized…
Most things have multiple functions. The tire iron you use to jack up the car when changing a flat tire? It can be used as a self-defense weapon if an…
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…
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…
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…
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…
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…
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