Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
This opinion piece was provided by Kevin Woodward of Phoenix, Maryland. Kevin is a member of the Barth Syndrome Foundation board of directors. The role of the U.S. Food and…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Kecia J, an…
On March 7, 2024, the United Kingdom’s Medicines and Healthcare products Regulatory Agency shared via press release that the agency has approved Ztalmy (ganaxolone) for people living with CDKL5 deficiency…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Prader-Willi syndrome (PWS) is a rare genetic disorder with a number of physical, emotional, mental, and behavioral manifestations. Individuals with PWS may exhibit cognitive impairment, hypotonia (poor muscle tone), and…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
A study led by a team of German scientists provides evidence of the safety and efficacy of CD19 CAR T cell therapy and its impact on autoimmune diseases. The study…
According to an article published over at Healio, a recent study has identified six biomarkers in rheumatoid arthritis patients that could predict cardiovascular disease. Prior research has demonstrated that people…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
According to a story from Healio, encouraging results from a phase 2 trial could herald a new standard of treatment for metabolic dysfunction-associated steatohepatitis (MASH). The investigational therapy is called…
The European Medicines Agency (EMA) explains that monepantel is an anthelmintic therapy, or a type of therapy that is used to expel worm-like parasites called helminths or nematodes from the gut.…
An 18-year-old woman in Korea underwent a kidney transplant after developing end-stage kidney disease, for which she had been treated with dialysis. Doctors were unsure of the cause. While the…
BioPharma Dive Source: Regenxbio data suggest ‘niche’ in Duchenne gene therapy
BioPharma Dive Source: Amylyx ALS drug fails crucial study, putting company’s future in doubt
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
There are many conditions which can be cured or at least ameliorated with a stem cell transplant or an organ transplant. But even when the right donor is available, and…
How one deals with adversity is very unique to the situation and the individual. I have faced great adversity at different times in my life, some of which I handled…
, organized by the EveryLife Foundation for Rare Diseases, is one of the most important annual advocacy events for the rare disease patient community. The event is held the last…
Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which donor immune cells attack the recipient's tissues. The graft cells recognize…
In February 2024, Jacob Bell reported in Biopharma Dive that pharmaceutical company Takeda Pharmaceuticals is planning on rapidly advancing late-stage testing of TAK-861 for people with type 1 narcolepsy (also…
In 2020, Chris and Susan Finazzo received news that would change the trajectory of their lives. They learned that their sons Chase (now 9) and Dylan (now 6) had a…
In early March 2024, Clinical Trials Arena reports that global biopharmaceutical company Bristol Myers Squibb shared new data from the Phase 3 DAYBREAK study. Within the study, researchers explored how…
According to a story from Medscape, a new set of guidelines from the Kidney Disease: Improving Global Outcomes (KDIGO) aims to improve and update standards of care for antineutrophilic cytoplasmic…
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