There are limited therapeutic interventions for those living with Niemann-Pick disease type C (NPC), a rare and progressive genetic disorder. Unfortunately, many people with NPC experience severe and life-threatening complications…
The Duchenne muscular dystrophy (DMD) community has a reason to celebrate. According to a recent news release, the FDA recently approved Duvyzat (givinostat) for people ages six and older who…
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…
Ronald Acuña Jr. is a professional baseball player with the Atlanta Braves. The powerhouse player is known for being an NL MVP and for becoming the first player in Major…
There are over 30 subtypes of congenital muscular dystrophy (CMD), or muscular dystrophies that are present at birth. These rare genetic disorders often manifest in hypotonia (low muscle tone) and…
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, advocates, scientists, and more.…
People with Cushing's disease have a benign pituitary tumor or pituitary growth that, when triggered, overproduced adrenocorticotropic hormone (ACTH). Too much ACTH causes the body to also overproduce cortisol. Known as…
Dupixent has long been known as a therapy which reduces itch, slows disease progression, and reduces exacerbations in a number of allergic conditions such as asthma, eczema, and nasal polyps.…
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, advocates, scientists, and more.…
Parkinson's disease is a progressive neurodegenerative disease. As neurons in the brain degenerate and die, those affected experience symptoms such as tremors or shaking in the hands, slowed movement, muscle…
Right now, no cure exists for people living with systemic sclerosis (also known as systemic scleroderma). There are treatment options available to manage systems. However, these also come with issues.…
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, advocates, scientists, and more.…
The Sudden Arrhythmia Death Syndromes (SADS) Foundation - a Patient Worthy partner - was founded in 1991 to provide support and care for people affected by heart arrhytmia conditions that…
The overwhelming majority of rare diseases and conditions have a genetic basis. This means that the disease is the result of a genetic abnormality such as a mutation. These abnormalities…
In the world of sports, the spotlight often shines on athletic prowess and competitive spirit. Yet Uplifting Athletes—a Patient Worthy partner—has transformed the narrative on the power of sport for…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Kristina Robinson, a…
For years, scientists have attempted to discover what destroys pneumococcal bacteria after a person has been vaccinated. In a recent study, vaccinologists in the U.S., China, and Switzerland, found the…
In early March 2024, Tyler Patchen reported that the U.S. Food and Drug Administration (FDA) approved both intravenous and subcutaneous formulations of Tyenne (tocilizumab-aazg) for use in a variety of…
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…
Rates of colorectal cancer are on the rise. In the US and other parts of the developed world, people seem to be developing this cancer earlier in life, for reasons…
Researchers worldwide spend years studying various aspects of a disease searching for a cure with help initially from public and private sectors. The primary source of the funding is usually…
Since 2015, the National Organization for Rare Disorders (NORD) has released an annual State Report Card that highlights state policies which could affect how people with rare diseases access care.…
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, advocates, scientists, and more.…
A majority of available treatment options for metachromatic leukodystrophy (MLD) rely on relieving symptoms. However, the recent approval of Lenmeldy (atidarsagene autotemcel) is the first ever FDA-approved gene therapy option…
When Josh Wright, 13, was younger, he always seemed to be somewhat of a sickly child. His mother Julie remembers Josh's first hospitalization at just one year old; her…
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