For over four decades, the aspiration of gene therapy has been to develop novel therapies with the potential to enhance human health. Recombinant AAV (rAAV), a type of gene therapy,…
In a recent report from Dermatology Times, Acelyrin has disclosed the disappointing outcome of its experimental therapy, izokibep, which failed to meet the primary endpoint in a phase 2b/3 clinical…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
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…
Early in the 1990s, anti-CD20 antibody therapy marked a significant improvement in the outcomes for patients with B-cell cancers. B-cell lymphoma is a type of cancer that originates in…
Currently, neoadjuvant chemotherapy is a key approach in treating ovarian cancer. Neoadjuvant chemotherapy aims to reduce tumor size, allowing for surgical removal following three cycles of chemotherapy. Additional chemotherapy…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
From September 7-9, 2023, the Rare Fair took place in Research Triangle Park, North Carolina, with the option for online attendance. This event, which originated in 2018, is organized by…
Acknowledgment: This story is sponsored by Janssen Pharmaceuticals and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted,…
Barrett's esophagus (BE) is a condition in which the cells in your esophagus undergo a transformation, becoming more like intestinal cells. Although doctors aren't entirely certain why this change…
Clostridioides difficile (C. diff) is a contagious bacterium that causes colon infections. People with C. diff infections (CDI) experience various symptoms, such as diarrhea and colonic inflammation, and in…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Clinical trials are essential for evaluating the safety, efficacy, and tolerability of various therapies, as well as their impact on the patient population. According to a recent news release…
This week Ionis Pharmaceuticals, Inc. issued a press release via PRNewswire announcing topline Phase 3 results for its Balance study of olezarsen. The study ( NCT04568434 ) enrolled people with…
Dupixent (dupilumab) is currently the first and only FDA-approved treatment for eosinophilic esophagitis (EoE) in people ages 12 or older who weigh at least 40 kg (88.18 pounds). The…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Sara Kear and Jake Kaufman always knew that they wanted to start a family. So they did, welcoming into the world their beautiful children: Lola, now age 3, and Mills,…
Rare Community Profiles 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,…
During her postdoctoral studies, Dr. Geneviève Bernard first discovered the gene responsible for 4H leukodystrophy (also known as 4H syndrome), a rare disease affecting the central nervous system. Now,…
In healthy individuals, copper is absorbed from food, and the excess is excreted by a substance called bile which is produced in the liver. It should be noted that…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Nasal polyps are non-cancerous growths that develop into the nasal passages. Generally, they don't cause any pain. However, when they reach a certain size, they can inhibit the sense of…
In 2001, Ebtesam Al Shehhi was 23 years old and looking forward to welcoming her first child into the world. When random symptoms began appearing, she first wondered if…
As reported in MedCity News, Ojjaara was part of GSK’s $1.9 billion acquisition of Sierra Oncology last year. FDA approval of GSK’s momelotinib covers the treatment of adult myelofibrosis…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
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