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…
According to a story from prnewswire.com, the experimental treatment SPR001 was just granted orphan drug status by the Food and Drug Administration (FDA). The new treatment is currently being developed…
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…
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…
Poor mental focus may be a contributing to the paralysis that commonly occurs after a stroke, according to a new study. A person who has had a stroke, when the…
WASHINGTON, D.C. & SAN DIEGO — Children’s National Health System and Retrophin, Inc. (NASDAQ: RTRX) are pleased to announce the creation of the Retrophin Rare Disease Network at Children’s National.…
The pharmaceutical company Protalix Biotherapeutics, Inc. announced that a drug currently in development called PRX-102 has been granted orphan drug status by the European Commission. The treatment is being developed…
The FDA passed along their nod of approval for the sought after gene therapy treatment, Luxturna, reported Engadget. This will be used to help aid those made blind by a…
Welcome back, Patient Worthians! We're starting out the new year with stories of strength and awareness. This week, we have an article about a man with CSF leak, whose life…
Music is sometimes more powerful than we even realize on the daily. Music keeps little 6-year-old Ollie Carroll going, even though he understands his life will be short. Ollie and…
In 2009 Emily Rochac Argueta realized her vision was rapidly deteriorating headed towards blindness, reported Times Union. The aspiring physician assistant was diagnosed with keratoconus, a rare eye disease that…
It's been 35-years since the passing of the Orphan Drug Act, and creation of the National Organization for Rare Disorders (NORD), reports PR Newswire. Even though these organizations recognize there…
Joy Littlesunday is just one of many Navajo newborns who were screened and thus saved from severe combined immunodeficiency by a successful bone marrow transplant at UC San Francisco, reported UCSF…
A new study suggests that the antidepressant medication clomipramine may function to relieve symptoms of multiple sclerosis. In this instance, the drug was tested to treat progressive multiple sclerosis, in…
Around 15,000 people are to receive free eye treatment thanks to the efforts of Senator Mustafa Bukar. The senator represents the Katsina North Senatorial Zone. Senator Bukar announced the event…
Maria Menounos's talent has graced the TV screen, American journalism and occasionally, the world of professional wrestling. With WWE, she serves as a hardcore ambassador to the company. She's a…
Happy New Years, Patient Worthians! New year, new you... new treatment news? This week, we have an article about a molecule that could change the way we treat cystic fibrosis.…
Pharmaceutical company, Santen, has been developing a drug, intravitreal sirolimus, to treat noninfectious posterior segment uveitis. Uveitis is a type of eye inflammation, in which the uvea, which is the middle part…
NORD, the National Organization for Rare Diseases, sent out their happiest wishes for the holidays, their utmost gratitude for 2017, as well as their plan of attack for the New…
After over a decade, Debbie Zelman, founder of Debbie's Dream Foundation, succumbed to her stomach cancer at age fifty. From the beginning, Debbie's prognosis was not good. When the cancer…
According to The Pharma Letter, over the last 20 years progress with the development of orphan drugs for rare diseases has been made, but many question, is it enough? There…
According to a story on PRNewswire, the biopharmaceutical company, Enzyvant, kicked off their Farber Disease Natural History Study in October. Acid ceramidase deficiency, also known as Farber disease, is an obscure…
When Avery was born, it didn't take long for her parents to realize that there was something different about her. She was not moving as much as most babies do,…
Just like any other 7-year-old little boy, Ethan Perkins has big dreams. He loves trucks and reading, and wants to study fossils as a paleontologist reported KXLY. He also wants to be…
Technology is hurting us. That's the claim made many times over the years. Smart phones may not have as many risks as some fear, but they can be a significant…
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