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…
The 2022 Medscape Physician Suicide Report shows that physicians, compared to the general public, have double the rate of suicides. Amna Shabbir, a doctor of internal medicine, felt compelled…
Indie songwriter and singer Sufjan Stevens recently posted on, Tumblr, his favorite media platform, that he has been diagnosed with Guillain-Barré syndrome (GBS). Sufjan is now in the hospital…
Tivdak, Seagen, and Genmab’s antibody-drug conjugate exceeded expectations. The partners said that a Data Monitoring Committee, independent of either company, noted that Tivdak’s overall survival rating met and even…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Previously, the FDA has approved Jardiance (empagliflozin) as a treatment option for adults with heart failure and to manage blood sugar in people ages 10+ with type 2 diabetes.…
From September 7-9, 2023, the Rare Fair was held in Research Triangle Park, North Carolina, and was also available for attendance online. This event began in 2018 and has been…
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