According to a story from Cystic Fibrosis News Today, Eloxx Pharmaceuticals had just announced recently that it is officially re-opening enrollment for its phase 2 clinical study. This trial will…
According to a story from PR Newswire, the US Food and Drug Administration (FDA) has recently approved a new treatment for Dravet syndrome, a rare disorder that causes seizures and…
Last week, my boyfriend took me camping for my birthday. But while we were out in the woods, he fell and injured himself; we thought his kneecap was dislocated…
According to a press release from HemoShear Therapeutics, the company has recently received official clearance from the US Food and Drug Administration (FDA) in regards to its Investigational New Drug…
The Rare Diseases Clinical Research Network (RDCRN) has recently launched a survey aiming to assess the impact of COVID-19 on the rare disease patient community. This initiative is important as…
According to a story from BioSpace, the biopharmaceutical company Dicerna Therapeutics, Inc. has announced recently that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to…
If you're like me, your life has changed immensely over the past few months. COVID-19, a novel coronavirus with 8.39 million diagnoses worldwide, has kept us inside and changed…
According to a story from Biotech 365, the biopharmaceutical company Deciphera Pharmaceuticals has recently announced that the company's drug ripretinib (marketed as QINLOCK™) has been approved by Health Canada. The…
There is now a new version of the Neupro patch coming! This generic version was created by Vektor Pharma. Vektor concentrates on transdermal systems both through patches on the skin and…
International Nonalcoholic Steatohepatitis (NASH) Day takes place on June 12th every year. This year, a 4 part podcast series was released by NASHNET as part of the continued effort to spread…
Antigen-specific T-cell therapy is an innovative treatment option which uses immune cells to combat cancer cells. AdventHealth is the very first in the world to use this treatment for patients…
During this time of COVID-19, many people have some general information about COvID-19 including the knowledge that individuals over 65 years of age are at higher risk of contracting…
Plakous Therapeutics released a press release to announce that Protego-PD™, their therapy for infants with necrotizing enterocolitis (NEC), received both the Orphan Drug and Rare Pediatric Disease designations from the FDA. There are…
According to Parkinson's News Today, the FDA is reviewing Gocovri as a potential treatment for "off episodes" in patients with Parkinson's disease who are already taking levodopa. "Off episodes"…
As reported in BioSpace; the US Food and Drug Administration (FDA) has granted breakthrough therapy designation, an expedited drug review, to brensocatib, a novel drug to treat non-cystic fibrosis bronchiectasis…
Leszek Ignatowicz, a researcher from Georgia State University, recently received a 5 year, almost 2 million dollar grant from the NIH for his investigation into multiple sclerosis (MS). This project…
June 19th is recognized as World Sickle Cell Day. This day is set aside to help spread awareness about sickle cell disease and its variants among the general public and…
According to a story from PR Newswire, the US Food and Drug Administration (FDA) has just approved a treatment for rare adult-onset Still's disease for the first time ever. The…
According to a story from Medpage Today, the US Food and Drug Administration has approved the drug lurbinectedin (marketed as Zepzelca), developed by Jazz Pharmaceuticals, as a treatment for metastatic…
Over 2018 and 2019, BioCryst Pharmaceuticals conducted two surveys to better understand the needs and expectations of hereditary angioedema (HAE) patients. Overall, they found that despite progress in HAE therapies,…
According to a story from Buzzfeed News, the results of a recent trial held in the UK may have finally revealed a medication that can have a major positive impact…
In the geographically small island nation of New Zealand, there are estimated to be as many as 300,000 people who live with various rare diseases or disorders. Unfortunately, like in…
June 17th is recognized as World CDKL5 Awareness Day and the month of June is considered CDKL5 Awareness Month. First organized by the International Foundation for CDKL5 Research (IFCR), the…
As reported in Globe News Wire, amidst a difficult time for rare disease patients as the medical world’s refocuses on the coronavirus, a new scholarship has been announced that could…
Celldex has just released positive results from a Phase 1 trial of an investigative therapy which could benefit patients living with chronic urticarias. This disease can result in decreased energy,…
© 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.
