In some cases, elderly patients (those over 70 years old) with esophageal cancer have been told that surgery is not the best option. This stems from a belief that…
According to a story from epmmagazine.com, a team of researchers from the Universities of Exeter and Nottingham have recently completely a study that could lead to the development of a…
Michael Conway, a grandfather from Scotland, believes that there should be increased rare disease screening after he was diagnosed with adrenoleukodystrophy (ALD) in 2016. Like many other rare disease patients,…
February 28th is known as Rare Disease Day, designed to raise awareness of rare diseases, patients, caregivers, and their experiences. As this day approaches, the American Kidney Fund (AKF) shared…
In 2007, biotechnology company Seagen Inc. and pharmaceutical company Astellas Pharma Inc. ("Astellas") collaborated to develop enfortumab vedotin. Now, in 2021, the pair submitted two supplemental Biologics License Applications (sBLAs)…
The Cystic Fibrosis Foundation is dedicated to finding a cure for the rare, genetic disorder. They fund more CF research than any other organization, and their latest effort is a…
According to a story from One News Page, the biopharmaceutical company BridgeBio Pharma and its affiliate ML Bio Solutions have recently announced that dosing has begun in its phase 2…
The FDA has recently accepted NeoImmuneTech's Investigational New Drug (IND) application for NT-17, a treatment for progressive multifocal leukoencephalopathy (PML). Now that the application has been cleared, the biopharmaceutical company…
During clinical development, treatments are sometimes first evaluated in healthy volunteers. This is the route that Insmed Incorporated ("Insmed") took with treprostinil palmitil inhalation powder (TPIP). Now, following positive topline…
After discovering that some pancreatic cancer expresses high amounts of intratumoral interferon signaling (IFN), researchers wondered whether they could create a new plan of treatment. According to Medical XPress,…
Recently, botanical pharmaceutical company Devonian Health Group Inc. ("Devonian") has been evaluating its product thykamine for patients with mild-to-moderate atopic dermatitis in a Phase 2 clinical trial. In a…
As reported in Biospace, Taysha Gene Therapies wants to eradicate devastating genetic epilepsies and central nervous system disorders rooted in a single genetic mutation. That’s going to take some innovative…
Biotechnology company Stealth Biotherapeutics ("Stealth") has made the company's mission to develop and commercialize treatments for patients with diseases characterized by mitochondrial dysfunction. One leading treatment in the company's…
According to a story from businesswire.com, the biopharmaceutical company Ipsen recently presented a total of 9 study abstracts at the recent 18th annual conference of the European Neuroendocrine Tumor Society…
According to a story from Sano Genetics, Sano Genetics, BenevolentAI, C4X Discovery, and Patients Know Best expect only the most from their novel collaboration. Together, they are pooling top expertise…
From February 25-27, the Americas Committee for Treatment and Research in Multiple Sclerosis will run its ACRTIMS Forum 2021, according to BioSpace. At this forum, Clene Inc. will present interim…
As many patients in the rare disease community know, achieving a diagnosis can be a lengthy and difficult journey. Following this journey, it can still take a while to receive…
Note: This guide was originally published on cysticfibrosis.com Written by Imogene The cystic fibrosis community is at high risk for serious complications from COVID-19. Through a recent survey of the…
A Phase 3 study of upadacitinib, an ulcerative colitis treatment, met both its primary and secondary endpoints, according to an announcement from AbbVie. The trial, titled U-ACCOMPLISH, saw a third…
Recently, clinical stage biopharmaceutical company Immunic, Inc. ("Immunic") has been working to develop potential treatment options for patients with primary sclerosing cholangitis (PSC), a rare condition for which no…
Research by scientists at Cornell University and Weill Cornell centered around the endothelial cells that form a barrier between tissues and vessels. They control the flow of fluid and substances…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
As the pandemic rages on, doctors across the country are beginning to notice a trend. A number of children who have contracted COVID-19 have also been impacted by a rare…
According to a story from ktvl.com, seven year old Emma Suetta, of Etna, California lives with a rare disease called cystinosis, and she is doing her part to help find…
If you’re a caregiver for a loved one with a rare disease, it’s not presumptive to assume you work tirelessly managing your loved one's busy appointment schedule, daily stretch routine,…
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