In a news release from biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. ("Alnylam"), the company shared that positive data was available from the Phase 3 APOLLO-B clinical trial.…
According to a story from Market Watch, the drug companies Merch and Eisai recently announced an update on their phase 3 clinical trial. This clinical trial was evaluating a combination…
According to a news release from biotechnology company Kira Pharmaceuticals, its therapy KP104 earned Orphan Drug designation in the United States. KP104 is in development as a therapeutic option…
According to a story from Myasthenia Gravis News, the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) has recommended ravulizumab (marketed as Ultomiris) to be approved as…
According to a story from Ophthalmology Times, the COVID-19 pandemic rapidly expanded the usage of telemedicine, including virtual, tele-ophthalmology appointments. It also highlighted the effectiveness of AI, and both of…
According to a story from Rett Syndrome News, the pharmaceutical company Acadia Pharmaceuticals is seeking U.S. Food and Drug Administration (FDA) approval for its investigational therapy trofinetide. It has been…
On July 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar for July. As always, these recurring meetings help provide critical updates to the rare disease community…
A recent article is bringing light to the condition known as misophonia, a disorder which causes people to have severe responses to certain sounds. Misophonia Misophonia, which literally translates…
Recently a woman was admitted to Odisha hospital where she was successfully treated for her rare blood disorder aplastic anemia. Aplastic Anemia Aplastic anemia is a rare, but serious,…
According to a story from ozarksfirst.com, Jamie Simoson was taken completely by surprise when her three year old son Jonathan started complaining about a headache. As his condition worsened, Jonathan…
The Powassan virus is extremely rare, however there have been cases of it in the United States. The disease is carried by ticks, but a recent article has pointed…
According to a story from Neuro Central, pharmaceutical company Merck has provided an update on its findings from its phase 3 clinical trial KEYNOTE-412. This clinical trial was evaluating pembrolizumab…
The HealthWell Foundation is a nonprofit organization with a mission to reduce financial barriers to care for underinsured patients with chronic or life-altering diseases. The foundation assists with copays,…
According to a story from Markets Insider, the drug company Kazia Therapeutics Limited recently announced that its investigational therapy paxalisib has been awarded Rare Pediatric Disease designation from the US…
Although many people in the United States face challenges when seeking healthcare services, new research has found that when seeking a diagnosis for a rare disease, the situation is…
When Stormi Vanden Bosch and Tyler Scotting found out they were expecting another son, they were thrilled. On October 31st, 2019, they welcomed their son Baylor to the world.…
The Catawba Virginia Farmers Market’s mission is to provide the Catawba community with local produce and handicrafts. Stephanie and Donald Schneider owners of Pacaberry Farms have been vendors at the…
For months, those within the rare disease community have been watching to see whether Upstaza (eladocagene exuparvovec) would be approved for use within the European Union. In fact, in…
To Diem Nguyen, her daughter Minh is her whole world. Diem would stop at nothing to ensure her daughter gets the help that she needs. That's why, recently, Diem…
In a recent news release, clinical-stage biotechnology company Amolyt Pharma ("Amolyt") shared that positive data was available from a proof-of-concept study evaluating AZP-3601 for hypoparathyroidism. Within this study, researchers sought…
When Eleni was first born, doctors and nurses told her parents, Neil and Eloise, that their daughter would most likely not survive. That’s because Eleni had been diagnosed with…
As time runs out, volunteer Dominic Gillen is running even harder. In an interview with KETV, he admits that they are not yet where they should be in order…
When Nicole Croxton and Pete Nappi first learned that they were going to have another daughter, the pair were thrilled. But during the course of Nicole’s pregnancy, doctors filled…
One thing is sure: 29-year-old Aimee Read was always a fighter. At just two years old, Aimee was diagnosed with leukemia. After two years of chemotherapy and six months…
According to a recent announcement, Epilepsy Research UK has announced a funding collaboration with the Tuberous Sclerosis Association which will support the investigation of tuberous sclerosis complex (TSC)-associated epilepsy.…
© 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.
