A groundbreaking clinical milestone has been reached in gastroenterology with the publication of a first-in-human study evaluating a novel spray gel for the treatment of ulcers following endoscopic submucosal dissection…
Applied Therapeutics has encountered a significant setback in its quest to bring a new treatment to patients with a rare metabolic disorder. According to Biospace.com, the company announced that its…
The use of the gene therapy elivaldogene autotemcel (Skysona), has been approved due to its success in the treatment of patients diagnosed with the neurological disease cerebral adrenoleukodystrophy (CALD). The…
According to a recent report in Healio News, the GLP-1 agonists are medications developed primarily to control blood sugar levels for type two diabetes. About the FLOW Clinical Trial The…
Spinal muscular atrophy (SMA) is a rare disease causing progressive muscular weakness. Severe forms of the disease can be fatal to infants. Apitegromab is an experimental therapy that was successful…
Written by Bill Clark Part 2 - Our Journey from Liver Failure to Transplant Continued from Part 1 “A new liver.” That was Becky’s answer pretty much every morning from…
Eighty-five-year-old Frank Meuers considers his 500th immunoglobulin infusion to be a cause for celebration. Frank has logged details of his doctor’s visits since his first dose of replacement therapy with…
Acknowledgment: This patient story is sponsored by Ipsen Biopharmaceuticals, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
The Riken Center team, headed by Dr. Takaomi Saido and using mice with Alzheimer’s, discovered that dopamine may lessen the brain’s physical symptoms and improve memory. The study, featured in…
It has been almost a decade since the European medical community welcomed news of an innovation for primary biliary cholangitis (PBC). Therefore, the EC’s recent conditional approval of Ipsen’s drug…
According to a story from Business Wire, the biopharmaceutical company Astria Therapeutics, Inc., recently announced that its experimental therapy navenibart has earned the US Food and Drug Administration's Orphan Drug…
According to a story from Healio, a recent study determined that adults living with Graves' disease who were being treated with antithyroid medications will have an elevated risk of developing…
A protein called Aplp1 is now on the radar for its ability to spread material in the brain that may be responsible for Parkinson’s disease. Another protein, Lag3, interacts with…
According to a story from Healio, analysis of the FDA Adverse Events Reporting System database indicates that tirzepatide has a superior safety profile when compared to GLP-1 inhibitors that have…
According to a press release from GlobeNewswire, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has granted the company's New Drug Application (NDA)…
Researchers discovered a version of the Cas protein that fits into adeno-associated viruses (AAVs). This virus can be engineered to deliver targeted therapy to cells and is a common approach…
According to a story from Business Wire, the biotechnology company Viridian Therapeutics, Inc., has completed its enrollment goal for its phase 3 clinical trial designated THRIVE-2. This clinical trial will…
According to a story from BioSpace, the biotech company Dianthus Therapeutics recently announced that the US Food and Drug Administration (FDA) has cleared the company's phase 2 clinical trial. This…
Women who undergo allogeneic hematopoietic cell transplantation (alloHCT) face significant treatment-related challenges regarding fertility. However, science seems to be overcoming many treatment-related fertility challenges after alloHCT and successful pregnancies are…
The COURSE Phase IIa clinical trial was conducted to evaluate tezepelumab (marketed as TEZSPIRE) in people living with chronic obstructive pulmonary disease (COPD) that had a wide range of blood…
In some cases, dermatomyositis can be difficult to treat. A variety of treatment options are available, from corticosteroids and immunosuppressants to antimalarials, colchicine, alendronate, and warfarin. But this rare condition…
The long-awaited positive results of Annexon Inc.’s Phase 3 trial investigating ANX005 for Guillain-Barré syndrome (GBS) was presented at the PNS 2024 Annual Meeting. Several leading global GBS experts spoke…
Yusara Ahmed was prepared to take action when she learned that her son Yusuf was diagnosed with beta thalassemia, an uncommon blood disorder. Yusara's sister also lived with the disease,…
According to a story from PR Newswire, the biotech company Mission Therapeutics was just awarded $5.2 million in funding to support MTX325, the company's investigational Parkinson's disease therapy. Intended to…
According to an article on Pharma Biz, Johnson & Johnson recently submitted a supplemental Biologics License Application to the US Food and Drug Administration (FDA). This application was for the…
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.
