As society becomes increasingly technological, researchers are getting closer and closer to mapping the entire human genome. In facts, shares Medical XPress, we may be nearly there. While many…
Researchers have been trying for years to better understand the mutations which cause the rare disorder called Charcot-Marie-Tooth disease (CMT). Tremendous progress has been made. But now that we know…
Today, American Gene Technologies (AGT) announced that they will soon be beginning a Phase 1 clinical trial for AGT103-T, a potential treatment for HIV. The unique gene therapy, developed…
Parkinson’s UK is nonprofit organization striving to help Parkinson's disease research continue amidst this global pandemic. Their goal is to get clinical research, and specifically studies that necessitate patients participating…
News-Medical Life Sciences recently interviewed Dr. Ananya Malhotra, Research Fellow at the London School of Hygiene, regarding her most current research involving pancreatic cancer. Dr. Malhotra believes artificial intelligence (AI)…
As reported by the National Hemophilia Foundation, researchers are seeking to understand what patients with chronic pain need and want, so as to make treatment safer and more effective. In…
Within six months of receiving funding from the Chan Zuckerberg Initiative (CZI), the TANGO2 Research Foundation (T2RF) is proud to announce funding of five new research projects and the hiring…
According to an article in Science Alert first published in the journal The Conversation, researchers at the University of Birmingham have shown that it is possible to prevent the…
At the virtual Alzheimer's Association International Conference (AAIC), AC Immune reported positive preclinical trial data for ACI-12589, their forward-thinking diagnostic tool. ACI-12589 is a non-invasive alpha-synuclein positron emission tomography…
In a recent press release, Swedish biotechnology company Active Biotech announced that the first patient in their Phase 1b/2a clinical trial received a dose of tasquinimod. The trial will…
When Jessica Kalnas was growing up, her mother Judy thought her daughter was just clumsy. The youngest of six kids, Jessica was always falling over: off of her bike,…
As published by the Food and Drug Administration; The U.S. FDA has just approved the first FDA approved therapy derived from cannabis (Epidiolex, a CBD solution) to treat tuberous sclerosis…
According to a story from BioSpace, the pharmaceutical company Marinus Pharmaceuticals, Inc. announced recently that its investigational product candidate ganaxolone has earned Rare Pediatric Disease designation from the US Food…
The HEALY ALS Platform Trial, being conducted by Biohaven Pharma, has begun enrolling patients. This study is intended to test the efficacy of three treatments on amyotrophic lateral sclerosis (ALS),…
CymaBay Therapeutics has recently released the results from ENHANCE, a study of seladelpar as a treatment for primary biliary cholangitis (PBC). These results were positive, proving that this medication has…
Alnylam Pharmaceuticals has submitted their clinical trial authorization (CTA) application to the Medicines and Healthcare Products Regulatory Agency (MHRA) for their RNAi therapy, ALN-HSD, a treatment for nonalcoholic steatohepatitis (NASH).…
According to Ryan McDonald of Cure Today, the FDA recently granted Breakthrough Therapy Designation to MK-6482, an investigational HIF-2α inhibitor developed by Merck. The orally-administered drug, designed to treat…
Within the last week, biotechnology company Levo Therapeutics announced results from the Phase 3 CARE-PWS clinical trial. During the trial, researchers examined the efficacy, safety, and tolerability of LV-101…
Rare disease or health-related foundations often encourage patient advocacy, research, and discussion around new treatments. According to Parkinson's News Today, the Parkinson's Foundation is doing their part in advancing…
Earlier this week, biotechnology company Time Technologies announced that its drug candidate SM-88 received Orphan Drug Designation from the FDA for the treatment of pancreatic cancer. The FDA defines…
Living with a rare disease can be difficult for a myriad of reasons, one of them being financial. Treatments, appointments, tests, and everything else that comes with a rare disease…
When Trey Kearns was two, he had a terrible itch. His itch wasn’t like yours - it was inside the body, meaning it's an itch you can’t scratch. They came…
Recently, biopharmaceutical company Vanda Pharmaceuticals announced that the FDA granted priority review status for a New Drug Application (NDA) and Supplemental New Drug Application (sNDA) for both liquid and…
According to a story from GlobeNewswire, the biopharmaceutical company CymaBay Therapeutics, Inc. recently announced encouraging topline findings from a phase 3 clinical trial. This study was testing seladelpar as a…
Just because we are staying home to avoid COVID-19 doesn't mean the medical field has stopped! In fact, many doctors are more determined than ever to improve patient outcomes.…
© 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.
