According to a story from Financial Buzz, the pharmaceutical company Proteostasis Therapeutics recently announced that the company's experimental treatment PTI-428 was granted Orphan Drug Designation by the FDA. PTI-428 is…
According to a story from pharmavoice.com, the pharmaceutical company Cadent Therapeutics has announced the beginning of the Phase I clinical study for its drug candidate CAD-1883. The therapy is a…
According to a story from EurekAlert!, scientists from UCLA have developed a new method of delivery for gene therapies called nanospears. These structures could allow for more accurate and precise…
Thanks to a local physician’s group in Baton Rouge, patients with amyotrophic lateral sclerosis (ALS) no longer face lengthy drives to receive treatment. The clinic serving the Baton Rouge area…
According to a story from BioSpace, the U.S. Food and Drug Administration has given Fast Track designation for nintedanib, which is undergoing testing for the treatment of systemic sclerosis with…
According to a story from raredr.com, The U.S. Food and Drug Administration (FDA) has recently approved Hizentra as a maintenance therapy for patients with chronic inflammatory demyelinating polyneuropathy. Hizentra is…
According to a story from raredr.com, the FDA had to order the cessation of Phase II/III trials for the microdystrophin transfer therapy SGT-001, which was in development for the treatment…
A “Right to Try” bill is currently being discussed by Congress. Advocates on both sides of the story present valid points to either pass or stop the bill. That’s why…
In a previous article we discussed what the Right to Try bill is, and what it’s current status was in the US Congress. It recently failed to pass in the…
A team lead by geneticist Christopher Gregg from the University of Utah conducted a study on elephant genes that discovered why elephants don't get cancer, reported by News 4 Tucson.…
According to a recent study, low copper levels could have a hand in leading to Charcot-Marie-Tooth disease, Charcot-Marie-Tooth News reports. Charcot-Marie-Tooth disease (CMT) is a neurological disorder that affects the…
Happy St. Paddy's everybody! While most people are putting on their green clothing to celebrate the holiday, some communities of rare patients have other exciting happenings in mind. This week,…
Shannon Hudson, mother from Columbus, is doing everything in her power to bring awareness to Glutaric Aciduria/Acidemia type 1, a rare disease her daughter was diagnosed with upon birth,…
The PTEN Hamartoma Tumor Syndrome Foundation is facilitating a patient symposium, themed "Knowledge is Power", March 25-26th in Huntsville, Alabama. The event will begin with a meet and greet at the…
According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…
Often children and other family members of patients with ALS end up being caregivers for their family, which can lead to isolation and a stressful life, reports WHYY. After Jodi…
When dealing with a rare disease, it's often hard to find someone else who's experiencing the same situation. Someone to talk with, cope with, listen to. Someone who gets it.…
According to a story from wired.com, Stephen Hawking, one of the most famous physicists on the planet, passed away on Wednesday, March 14th, 2018. He was 76 years old. His…
According to a story from All4Women, Nicole Capper, crowned Mrs. South Africa, is planning to summit Mt. Everest, the highest mountain peak on the planet, in order to help promote…
Forty-eight-year-old Isabel lives with the genital skin condition called lichen sclerosus, being diagnosed post menopause, and it's not an easy new life-style, reported NetDoctor. Lichen sclerosus is a rare genital…
The BIOIMAGING 2018 Conference in Portugal recently announced it’s “Best Student Paper” award. The award went to a Nigerian doctoral student who identified a new technique for examining the retina.…
Last week was World Kidney Day! And in fact, the whole month of March is kidney month - so there's no excuse to brush up on some key kidney facts…
NEW YORK (PRWEB) MARCH 13, 2018 – The American Liver Foundation (ALF), the nation’s largest patient advocacy organization for people with liver disease, announced today the launch of an online…
Scientists in California at Salk Institute for Biological Studies are currently developing a drug that can mimic the effects in the body usually obtained from exercise, reports the National Post.…
According to a story from the Wall Street Journal, the U.S. healthcare system might be able to learn a thing or two from Netflix. A well understood aspect of healthcare…
© Copyright 2024 Patient Worthy
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.
