Even though August is officially Psoriasis Awareness Month, Healthline recently put together a list of the top online advocates in the psoriasis community that are bringing awareness all year. Here…
Batten disease is one of the rarest diseases in the world. In fact, it only affects about two to four out of every 100,000 births in the United States, which…
Recent findings from research done at the University of Pennslyvania indicate a new biomarker associated with a group of rare metabolic diseases called mucopolysaccharidoses (MPS). A biomarker is a measurable…
A North Carolina-based research group has just developed research that indicates a potential stem cell treatment for lung fibrosis conditions such as cystic fibrosis (CF) and idiopathic pulmonary fibrosis (IPF).…
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…
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…
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…
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…
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…
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…
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…
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!…
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:…
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…
Organ donation is an intensive process to go through. It takes a ton of time to find a match, and even after the donation has taken place, recipients run a…
Soleno Therapeutics, Inc. of Capnia reported in early July of this year that the company plans to start a Phase 3 clinical trial for evaluating diazoxide choline controlled-release (DCCR) in…
The latest in Waldenstrom macroglobulinemia news comes not only from the hospital clinic, but also from the race track, and the dance floor! As for the former, latest results are…
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