According to a story from pharmiweb.com, the pharmaceutical company Ovid Therapeutics announced recently that the U.S. Food and Drug Administration (FDA) has given its drug candidate OV101 Fast Track designation…
According to a story from aninews.in, mother Shashi Mishra and her daughter Anamika Mishra have staged a hunger strike in a desperate move to receive treatment for their muscular dystrophy.…
According to a story from engadget.com, the tech company AliveCor, known for its KardiaBand device for Apple Watch, is currently developing new capabilities for its innovative watch strap. Currently, the…
According to a story from sciencedaily.com, new and improved standards of treatment are allowing patients with Duchenne muscular dystrophy to have longer lifespans than in the past. By all accounts,…
According to a story from the Herald Sun, Matilda Mahon, an eighteen month old girl from Australia, will need to travel to the United States in order to receive treatment…
26-year-old Rebecca Smith's pregnancy was going as smooth as can be until she was 8-months pregnant, reported Hertfordshire Mercury. Rebecca discovered a lump on her neck and went in for…
Just recently, MMJ International Holdings announced that MMJ Bioscience has received proper Canadian licensing to grow cannabis specifically to develop cannabinoid medicines to treat patients battling multiple sclerosis and Huntington's…
According to a story from Xconomy National, some patient advocacy organizations are beginning to speak out about exorbitantly high drug prices despite the fact that many of them receive funding…
According to a story from The European Society For Medical Oncology, a recent study shows that young adults and teenagers are less likely to adhere to follow-up guidelines following treatment…
According to a story from guttmacher.org, the U.S. Department of Health and Human Services proposed a host of new regulations purported to enforce over 20 federal provisions related to "conscience…
According to a story from PR Newswire, the pharmaceutical companies Sanofi and and Regeneron Pharmaceuticals are implementing a plan to make Praluent injections more accessible and affordable for patients with…
The apparent goal behind the current Right to Try legislation is to create better access to life saving medication. Many people are excited about the possibilities such a bill could…
According to a story from pharmpro.com, the pharmaceutical company Abeona Therapeutics announced that it has been given Rare Pediatric Disease Designation for its ABO-202 program by the U.S. Food and…
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,…
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