The 20th annual WORLDSymposium took place from February 4-9, 2024. During the course of the research conference, at least one presentation centered on data from the Phase 2 Mini-COMET long-term…
The 17th Annual Congress of the European Association for Haemophilia and Allied Disorders took place this year from February 6-9, 2024. During the Congress, stakeholders came together to discuss how…
Imagine if you had recurring attacks of severe and unexplained swelling beneath your skin. For individuals living with hereditary angioedema (HAE), a rare inherited disorder, this isn't just a mere…
Metabolic dysfunction-associated steatohepatitis (MASH) unfortunately has no treatments to reverse the disease or its damage. MASH can be managed through weight loss, regular exercise, and blood sugar management. But identifying…
Despite novel treatment advances, hepatocellular carcinoma (HCC) remains difficult to treat. This aggressive cancer is often not found until later stages and not all people with HCC can utilize available…
According to reporting from Healio Psoriatic Disease, the Food and Drug Administration (FDA) recently updated the label for Dupixent (dupilumab) in the indication of moderate-to-severe atopic dermatitis with moderate-to-severe hand…
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.…
The American Society of Clinical Oncology (ASCO) held its ASCO Gastrointestinal Cancers Symposium from January 18-20, 2024. During the Symposium, doctors, researchers, scientists, and other stakeholders discussed innovative science and…
Chronic spontaneous urticaria (hives) - an itch that can't be scratched. Well, from a literal perspective, you can scratch. But the chronic nature of chronic spontaneous urticaria means that your hives will…
In December 2023, pharmaceutical and biotechnology company Moderna announced that results from a Phase 2/3 clinical trial were published in The New England Journal of Medicine. The study explored an mRNA-based…
In December 2023, clinical-stage biopharmaceutical company Clene Nanomedicine ("Clene") shared that it had met with the U.S. Food and Drug Administration (FDA) regarding Accelerated Approval for CNM-Au8®, an experimental therapy…
In the United States, companies must obtain approval from the U.S. Food and Drug Administration to run clinical studies and transport or distribute therapies across state lines. To do so,…
In the past, Hyqvia [immune globulin infusion 10% [human] with recombinant human hyaluronidase] was approved to treat people with primary immunodeficiencies (PIs) ages two and older. Now, following data from…
Unfortunately, clinical studies do not always work out the way they’re expected to. Biopharmaceutical company Mirum Pharmaceuticals, Inc. (“Mirum”) learned this after the Phase 2 EMBARK trial failed to meet…
A drug cannot be transported or distributed across state lines until its marketing application has been approved/cleared, so getting this clearance is crucial for many drug developers and physicians who…
AIV001 (axitinib): a potential, non-surgical therapeutic option for people with basal cell carcinoma (BCC). AiViva Biopharma describes AIV001 as: a novel formulation of a multi-kinase inhibitor combined with AiViva’s…
In the United States, Breakthrough Therapy designation is granted by the U.S. Food and Drug Administration (FDA) to expedite drug development and review. This designation is granted to therapies that…
In an investor news release from December 2023, Moderna and Merck shared follow-up data from the Phase 2b KEYNOTE-492/mRNA-4157-P201 clinical trial. During the trial, researchers explored the impact of Keytruda…
In December 2023, Merck and HiberCell announced that the organizations would be partnering on a Phase 1b clinical study to examine a drug combination for clear cell renal cell carcinoma…
Have you ever heard of Orphan Drug designation? This designation, granted by the U.S. Food and Drug Administration (FDA), is given to drugs or biologics that are designed to treat,…
Right now, there are minimal therapeutic interventions for people living with idiopathic pulmonary fibrosis (IPF). Because of this, IPF comes with a life expectancy of 3-5 years past diagnosis.…
Clinical trials are helpful tools in deepening our understanding of certain diseases and evaluating and identifying effective treatment options. Unfortunately, these trials don't always garner the necessary information. For…
Last year, the FDA placed a clinical hold on an Investigational New Drug (IND) application for deucrictibant for the on-demand and prophylactic treatment of individuals living with hereditary angioedema…
Over their lifetime, people with neurofibromatosis type 1 (NF1), a rare genetic disorder, have a 30-50% chance of developing plexiform neurofibromas. These tumors grow on peripheral nerve sheaths throughout…
The risk of developing an inflammatory bowel disease (IBD) such as Crohn's disease or ulcerative colitis (UC) has been slowly but steadily increasing over time, with an estimated 0.8%…
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