.jpg)
A molecular biologist, Dr. Daria Julkowska, coordinates an international consortium in Paris that is largely funded through the 28-member European Union (EU). According to a report published in the Charcot-Marie-Tooth…
According to a publication from Augusta University, a recent study lung cancer patients receiving chemotherapy provided a possible explanation for some cancers' ability to resist treatment. The research, conducted by…
According to a story from Angioedema News, a recent study demonstrated the effectiveness of the medication Haegarda in reducing the number of swelling attacks experienced by hereditary angioedema patients. Preventative…
According to a story from Myeloma Crowd Crowdcare Foundation, the very first Plasma Cell Disorder Congress recently took place in Charlotte, NC from August 10-11, 2019. The event primary focused…
According to a story from ProPublica, the computer servers that store important patient medical data are surprisingly unprotected. This lack of protection appears to be a problem around the world,…
According to a story from globenewswire.com, the gene and cell therapy company Abeona Therapeutics Inc. recently announced that has received a letter from the US Food and Drug Administration (FDA)…
According to a post from gaucherdisease.org, clinical trials are an essential component to the development and testing of new therapies for Gaucher disease and other rare diseases. For some rare…
.jpg)
Alzheimer’s disease (AD) is a neurodegenerative disease and the major cause of impaired memory affecting the older population. Reported cases are rapidly increasing. So far, clinical trials have failed…
.jpg)
The accelerated approval granted by the FDA for the Duchenne muscular dystrophy drug eteplirsen, developed by Sarepta Therapeutics, was granted only after internal disagreements and protests had been resolved.…
A minimum of seven people have died after contracting Eastern Equine Encephalitis (EEE), a rare disease carried by mosquitoes, bringing number of total people affected to 27 in six states.…
September 25th is International Ataxia Awareness Day! The National Ataxia Foundation (NAF) has great resources to help spread the word and show our solidarity for the Ataxia patient community; on…
.png)
Fierce Biotech recently focused on a report by The BMJ, a British science journal, on the validity of clinical trials which are critical to decision making by patients and…
Tay-Sachs Disease September is Tay-Sachs Disease Awareness Month. Voted on by the Senate unanimously in 2008, this month aims to spread awareness about this disease and the importance of carrier…
According to a recent publication from the Duke University School of Medicine, Dr. Rodger Liddle believes Parkinson's disease may have origins in an unlikely place — the gut. Liddle, a…
According to a story from delmarvanow.com, a nine year old boy named Trey Kearns has progressive familial intrahepatic cholestasis (PFIC) a rare disease that affects the liver. The symptom that…
September 24th is Familial Hypercholesterolemia (FH) Awareness Day! Let's take a closer look at FH, and how we can help make this day productive for the FH -- and rare…
According to a story from scienmag.com, a pair of five year grants that were awarded to Dr. Mark Zylka will go towards projects focused on autism and the rare disease…
According to a story from the Washington Post, Bob Schwartz is a walking, talking medical mystery. He lives with a strange and diverse array of difficult symptoms, and after being…
Outcomes of a pilot study on Gaucher disease using a mobile phone app and wearable technology demonstrate a more accurate approach to disease assessment. Gaucher disease is a lysosomal storage…
According to a story from Benzinga, the medical device company V-Wave Ltd. has recently announced that a product in development by the company has earned Breakthrough Device designation from the…
According to a story from clinicaltrials.gov, the Children's Hospital of Philadelphia is sponsoring a study that has the potential to make mitochondrial disease research and drug development much easier. The…
Genes themselves are a relatively new discovery, found only about a century ago. Gene therapy, a medical procedure that alters genes to address disease, is a concept a few decades…
According to a press release from the California-based biotechnology company Ardelyx, the US Food and Drug Administration (FDA) recently approved the company's irritable bowel syndrome drug candidate Ibsrela (generic name…
A recent publication from BMC Pediatrics outlines a case study of Kawasaki disease in a group of infants from Italy. Because Kawasaki disease is a rare disease (with only an…
According to a press release from CTD Holdings, the American Food and Drug Administration (FDA) recently approved an Expanded Access application from a physician to treat a pediatric Niemann-Pick disease…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
