On October 24, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
Only around 350 to 500 people in the United States are diagnosed with Creutzfeldt-Jakob disease (CJD) each year. Unfortunately, this disease often causes rapid cognitive decline and physical deterioration,…
According to a story from Healio, 21 people living with the disease prurigo nodularis took part in a qualitative study over the phone in which they shared the ways in…
Currently, there are no FDA-approved treatments designed for cognitive impairment related to Huntington’s disease. However, to improve the lives of those affected, additional research and therapeutic development are urgently…
The two trials evaluating lacutamab, an investigational therapy developed to treat T-cell lymphomas, were put on partial hold by the FDA. The patient died of hemophagocytic lymphohistiocytosis, which is…
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…
In 2018, the U.S. Food and Drug Administration (FDA) approved Onpattro (patisiran) as a treatment for polyneuropathy in people with hereditary transthyretin-mediated (ATTR) amyloidosis. Onpattro is an intravenously administered,…
Genomics is revolutionizing healthcare processes, offering the potential to enhance the lives of numerous individuals by enabling the early detection of treatable disorders and providing lifesaving therapies. Every year, thousands…
According to a story from Healio, the investigational therapy sotatercept has been given Priority Review status from the US Food and Drug Administration (FDA). The therapy is in development as…
Patient Worthy partner Alex TLC has announced that, in a large-scale newborn screening study supported by the UK's NHS and Genomics England, adrenoleukodystrophy (ALD) and metachromatic leukodystrophy (MLD) will be…
₹17.5 crores—or $2,102,128. That's the cost of medication for a young boy named Kanav, who lives in Najafgarh, a town in the Southwest Delhi district. Kanav is living with…
After years of seeking approval for the treatment of Pompe disease, this week, as reported by GlobeNewswire, the FDA granted approval to its developer, Amicus Therapeutics, for the first…
This week, the USFDA granted a new marketing authorization for the ICHC Cancers Panel, thereby creating a new regulatory classification. According to a report in Inside Precision Medicine, the…
Interferon beta was the first available therapy for treating multiple sclerosis (MS) and is still the most commonly prescribed treatment. This therapy helps reduce relapses in MS patients. Distinguished UCLA…
7-year-old Isla Edwards suddenly found herself struggling to see. Her vision became fuzzy when trying to focus on objects at a distance. Her parents assumed this meant Isla needed…
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…
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…
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…
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…
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…
You are invited to join hundreds of thousands of people touched by spina bifida, the most common congenital birth defect causing disability. There is no cure. This is a…
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