Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…
Every three to four minutes, someone in the U.S. is diagnosed with a blood disease such as lymphoma, leukemia, or sickle cell anemia. As reported in the Pittsburgh Post-Gazette,…
Chip Wilson, billionaire founder of Lululemon Athletica, was first diagnosed with muscular dystrophy in 1987 at the age of 32. Wilson, who is now 66 years old told Global…
In 2010, Eddie Vedder (well known for his musical career in Pearl Jam) co-founded the EB Research Partnership (EBRP) alongside wife Jill and a group of parents whose children…
In 2005, the U.S. Food and Drug Administration (FDA) approved abatacept for the treatment of moderate-to-severe rheumatoid arthritis. Twelve years later, in 2017, the FDA expanded the drug's approval…
Arcus Biosciences and Gilead Sciences issued a press release announcing encouraging overall response rates and six-month progression-free survival rate for their combination treatment of domvanalimab plus zimberelimab and chemotherapy…
In the past, the U.S. Food and Drug Administration (FDA) approved Opdivo (nivolumab) for different forms of cancer, such as non-small cell lung cancer (NSCLC) and melanoma. More recently,…
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…
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…
Imagine the frustration of Faustina Cavero, unable to follow her maternal instincts that told her something was wrong with her baby. Doctors at two different hospitals disagreed with her and…
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…
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…
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