RespireRx Pharmaceuticals announced last month it was cleared to resume its ampakine CX717 clinical trials to evaluate the drug’s efficacy in several central nervous system and respiratory disorders, including Pompe…
Last month, the FDA has granted Achillion Pharmaceuticals orphan drug designation to ACH-4471 for the treatment of patients with C3 Glomerulopathy (C3G). According to the FDA, the Orphan Drug Designation program provides…
New research is helping experts understand the underlying causes of idiopathic pulmonary fibrosis on the molecular scale. When solving any problem, disease included, it is best to start by understanding…
Nothing is worse for a family than hearing the word “cancer,” especially when it involves a child. The Tyson family was ready to start their perfect life eleven years ago.…
According to a story from the National Hemophilia Foundation, the Swedish pharmaceutical company Orphan Biovitrum announced that its first patient was registered for a new phase 4 clinical trial. The…
Another successful partnership in the rare disease community has come to fruition, reports The Pharma Letter. Just this past week, CureVac and Arcturus Therapeutics will become a powerhouse together with…
Happy Friday, Patient Worthians! This week we have articles about people working together on different types of rare disease teams. We have stories from a family who supports two children…
Patient Worthy is proud to announce we are participating in Patients as Partners EU in London, UK on January 23rd. Patients as Partners EU represents the only conference opportunity in Europe…
Margarita Matias could not believe her eyes when she walked into the newly renovated bathroom. Her old bathroom was far from ideal, and often leaked. The new arrangement is optimized…
According to an article from businesswire.com, the pharmaceutical company Odonate Therapeutics announced the beginning of CONTESSA, a phase three study of the treatment tesetaxel and its function in patients with…
Patients suffering from sickle cell disease, SCD, might just be getting news they've been waiting to hear for a long time. Just this week, the U.S. Food and Drug Administration…
At three months old, Emily's mouth hurt. Her first tooth started coming in. Her parents immediately knew that something was wrong. Three months later, the family received dire news about…
While it's general knowledge that working out is a huge plus for a healthy life, many questioned whether it was positive or dangerous to individuals with rare diseases. Yet, BMC…
According to a story from philly.com, many people with severe forms of brain cancer are not receiving sufficient hospice treatment, according to a recent assessment. The study took data from…
According to a story from financialbuzz.com, the pharmaceutical company AveXis, Inc., is planning to begin preparations to submit a Biologics License Application (BLA) to the Food and Drug Administration for…
According to a story from the Chicago Tribune, Ben Barres, a widely renowned neurobiologist and researcher, passed away at age sixty-three after fighting pancreatic cancer. Mr. Barres was credited with…
5-month-old baby, Rohaan's, life was spared when he recently underwent surgery to treat his rare congenital heart condition, surgery that almost didn't happen, reported Times Of India. His parents, Muhammad…
Watch Prof. Charles Craddock discuss important and heartening results he and his team presented at the American Society of Hematology 2017 Annual Meeting and Exposition in Atlanta, GA - regarding whether there…
When you are in the midst of difficult times in your life, what do you do to make it through? Do you try and find a way to hide out…
2017 was a challenging year for 10-year-old Zach Pickard, but at first glance of his smile and rolling laughter, you would never expect it. Zach is among 144 children in…
Cycle Pharmaceuticals shook hands with US-based firm, Aprecia Pharmaceuticals as they agreed to develop and commercialize treatments for rare diseases using the advanced technology of 3D printing, recently reported Cambridge…
Gene therapy has successfully made its presence known in the medical field this last year, reported ABC News. Dr. Francis Collins of the National Institutes of Health isn't afraid to…
A recent study has revealed a possible mechanism for the repair of nerves damaged by multiple sclerosis. Research at the University of California has found that gene expression in certain…
Organovo Holdings recently stated that its 3D bioprinted therapeutic liver tissue was granted orphan drug status by the Food and Drug Administration (FDA). Taylor Crouch, the company CEO, was excited…
The world of rare disease is seeing a ray of sunshine with a viable partnership. Two powerhouses of patient advocacy are uniting together to bring about change, awareness and cure-searching.…
© 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.
