It's a good time to check in on your eye health because January is Glaucoma Awareness Month! More than 3 million people in the United States have glaucoma - and…
January is Thyroid Awareness Month! It may be difficult to find time to learn a little more about your body and health tips - but since it's Thyroid Awareness Month,…
January is National Birth Defects Prevention Month! A birth defect is defined as a problem that happens while a baby is developing in the mother's body. Most birth defects happen during the…
Tomorrow is Moebius Syndrome Awareness Day! Moebius Syndrome Awareness is celebrated globally each year - with the goal of raising awareness about this rare condition and to educate the many…
Days in 2018, we have some great statistics on 2017! According to the United Network for Organ Sharing (UNOS), over 10,000 organ donations were made, marking the first year that number has been…
The Loma Linda University Eye Institute in California performed its first-ever procedure using a FDA-approved procedure, which can deter the effects of keratoconus - a rare progressive disease of the cornea,…
As a wave of female empowerment cascades our nation - like the Time's Up and #MeToo movements - let's also turn our attention to Cervical Health Awareness Month. Cervical Health…
Although it never should have happened, at least the right course of action was taken! Reggie Bibbs of Houston, Texas was turned away from his Uber driver when he requested…
REGENXBIO announced last month that the Investigational New Drug application (IND) is active for a Phase I/II clinical trial of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II)…
RespireRx Pharmaceuticals announced last month it was cleared to resume its ampakine CX717 clinical trials to evaluate the drug’s efficacy in several central nervous system and respiratory disorders, including Pompe…
Last month, the FDA has granted Achillion Pharmaceuticals orphan drug designation to ACH-4471 for the treatment of patients with C3 Glomerulopathy (C3G). According to the FDA, the Orphan Drug Designation program provides…
Watch Prof. Charles Craddock discuss important and heartening results he and his team presented at the American Society of Hematology 2017 Annual Meeting and Exposition in Atlanta, GA - regarding whether there…
One of the medical research field's biggest developments recently - within the last 5 years! - is a new form of gene editing, also know as genome editing. Gene editing refers to…
Irish drugmaker, Shire, announced earlier this month it will bring new genetic disorder drugs to India, stepping up its worldwide presence. Shire is unique in that it's one of the…
Kei Otake is a 19-year-old cello student at The Juilliard School - a prestigious performing arts college in New York City that boasts alumni like Viola Davis, Robin Williams, Adam Driver…
The Washington Post published a bizarre story earlier this month that is equal parts interesting and terrifying! A US National Security Agency counterintelligence officer (AKA a spy) is claiming that while…
A video of former US Vice President Joe Biden on the talk show The View comforting co-host Meghan McCain has been making the rounds on the Internet the last few…
The FDA has granted a special designation for a drug company developing a gene therapy for the treatment of leber congenital amaurosis (LCA). MeiraGTx received rare pediatric disease designation for its…
During a time when we get a lot of bad or polarizing news about the world, it's important to highlight the good stuff - so here is some good stuff…
University of California, San Francisco scientists have developed a new vaccine that treats diffuse intrinsic pontine glioma (DIPG), a rare childhood brain tumor. To learn more about DIPG, click here. Great…
Great news for the rare disease community! Ultragenyx Pharmaceuticals reported back positive results from its phase 3 study of the treatment drug burosumab in adults with X-linked hypophosphatemia (XLH). XLH…
I want to be Isaiah Acosta when I grow up. The 17 year-old from Phoenix, Arizona was born without a jaw, due to the very rare disease situs inversus, which…
The Leukemia Research medical journal published results from a study that showed efficacy when treating Acute Myeloid Leukemia (AML). Great news for the rare disease community! Onconova Therapeutics announced the publication of results…
The HLN channel aired a documentary special regarding the rare and mysterious condition, fatal familial insomnia (FFI). FFI is a very rare genetic sleep disorder, with less than 40 families diagnosed…
A major first step for a viable treatment option! Achillion Pharmaceuticals last week reported preliminary proof-of-concept results from its ongoing Phase 2, study of a new treatment for C3 glomerulopathy…
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