Sometimes, it can be difficult to incentivize researchers and drug developers to focus on solutions for those with rare conditions. The FDA created the Orphan Drug Act to overcome…
Through its recently issued press release, bluebird bio announced the FDA’s accelerated approval of the first therapy to slow the progression of adrenoleukodystrophy (ALD). The disease is the result…
Are you ready for the October 2022 Dysautonomia Challenge? You can make this October stand out on your calendar by making each day meaningful, event ladened, exciting, and dedicated…
In April 2022, the United States approved Ultomiris for the treatment of patients with generalized myasthenia gravis (gMG). This approval was followed, just four months later, by an approval…
According to a story from Undark, Renee Schmidt's first signs of Ehlers-Danlos syndrome became really noticeable when she was a freshman in college. She would experience brief bouts of memory…
Patient Worthy is proud to announce that we have recently partnered with Life on the Level, a support group and advocacy organization based in Leicestershire, United Kingdom. Life on the…
From October 3-21, 2022, HAE Junior, a Patient Worthy partner organization, will have its art exhibit on display in the halls of the Czech Parliament. HAE Junior is an advocacy…
The new nonprofit Every Cure has been created on the basis that every drug currently on the market effects multiple areas in the body. This premise has been proven…
According to a story from the Glasgow Times, David Steed of Paisley, UK, was diagnosed with adrenoleukodystrophy when he was 18 years old. At the time, he didn't have any…
In his early stages of life, Austin (A.J.) Rose was diagnosed with an ultra-rare disease called pontocerebellar hypoplasia type 6 (PCH6). Now, at three years old, A.J. is a…
In late September 2022, biotech company Oncoheroes Biosciences, Inc. ("Oncoheroes") shared via news release that its product dovitinib, which the company has an exclusive pediatric licensing agreement for from…
According to a story from Yahoo! News, a mother that is well attuned to her children can often have a special intuition when something is wrong. Thirteen year old Sheldon…
According to a press release, the gene therapy company bluebird bio, inc. has recently announced that its gene therapy elivaldogene autotemcel (marketed as SKYSONA) has received Accelerated Approval status from…
According to an article from Cancer Network, the U.S. Food and Drug Administration (FDA) recently granted Orphan Drug designation to second-line GNS561 (ezurpimtrostrat) for cholangiocarcinoma (bile duct cancer). In…
Jim Kelly and his wife Maria still remember the day they received a call from the hospital telling them that according to test results, their daughter Adriana, now age…
A New Theory The Week reports that scientists at the Krembil Brain Institute in Toronto are working on a new theory about the origins of Alzheimer's disease. The Toronto…
According to a story from neurology.duke.edu, Duke University has recently been recognized as a Center of Excellence for Dystonia treatment by the Tyler's Hope for a Dystonia Cure Foundation. This…
Did you know that September is National Sickle Cell Awareness Month? During this month, various stakeholders come together to share the stories of those affected by sickle cell disease,…
The Orphan Drug Act of 1983 was designed to facilitate the development of orphan drugs, or drugs designed for patients with rare or life-threatening conditions. Now, the FDA grants…
ScienceAlert recently published an article explaining how scientists at Rice University have been able to eradicate tumors in mice by using a new treatment that takes less than a…
September 24, 2022 will be recognized as Familial Hypercholesterolemia (FH) Awareness Day, a time to spread awareness about this under-recognized condition among the general public and in the medical field.…
Joe and Cynthia are the founders of Jordan's Guardian Angels, a Patient Worthy partner organization that is committed to supporting research into Jordan's syndrome, an incredibly rare disorder named after…
According to a story from UN News, the World Health Organization (WHO), in conjunction with partners, has initiated a new vaccination campaign targeting meningitis in Africa. This is part of…
According to a story from Buzzfeed News, actor and activist Jane Fonda, age 84, recently announced that she was diagnosed with a form of non-Hodgkin lymphoma. She has begun treating…
The 1st International Workshop on Myelodysplastic Syndromes was held this year in Miami, FL from June 24-26. This in-person event was a convergence of researchers and clinicians to discuss advances…
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