Written by Sara Tompkins NOTE: The perspectives voiced in this article are solely those of the author. The development of drugs for rare diseases has always been a complex and…
From September 19-21, 2023, the Global Genes RARE Advocacy Summit was held in San Diego, CA. This event is a yearly convergence for rare disease stakeholders with a special emphasis…
If you have read my books, seen my blogs, or watched my videos, you know that I live with a neurological movement disorder called dystonia, which I've had since 2001.…
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…
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…
Newborn screening is an important public health initiative that checks for rare and serious genetic, blood, hormonal, or metabolic conditions at birth. The test collects blood through a heel…
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…
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…
Editor's Note: Chronic conditions and rare diseases don't discriminate. Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
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…
₹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…
After around a 10-month hiatus, we are excited to announce that Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back in action with…
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…
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…
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…
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,…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
When young Emmett Cordes initially complained of knee pain, his mother attributed it to his active nature. However, as the pain persisted, a visit to the doctor and subsequent…
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…
Advancing patient outcomes hinges on the development of targeted treatments, particularly in conditions with grim prognoses such as small cell lung cancer (SCLC), which typically has a life expectancy…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
