According to a story from azfamily.com, Reyna Felix, age 28, was at work as a 911 dispatcher when her coworkers said that she started behaving strangely. Around midnight, she fell…
An estimated 1 in 10 Americans, or 1 in 20 people globally, have or will have a rare disease at some point. Rare Disease Day takes place on February 28. Ultimately,…
According to a February 25 press release, Amondys 45 (casimersen), a therapeutic option for patients with Duchenne muscular dystrophy (DMD), recently became FDA-approved. The therapy is specifically designed for patients…
On February 18, 2021, specialty pharmaceutical company Kyowa Kirin Co., Ltd. ("Kyowa Kirin") shared that a Phase 2 clinical trial evaluating KHK4083 had reached its primary endpoint. KHK4083 is an…
According to BioSpace, Biohaven Pharma has utilized their MATE platform to create and develop a hyperimmune globulin mimic (HGM) that is able to both bind to and neutralize various strains…
Billions of dollars have been spent in research over decades, resulting in only two therapies that treat symptoms of Alzheimer’s disease (AD). There are no approved methods that diminish the…
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…
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