Having a rare disease, or a family member with a rare disease, can be expensive. Management often requires multiple visits to specialists. Medication and medical equipment are not always covered…
You may not have heard of spinocerebellar ataxia 4 (SCA4). It is rare and it is a devastating movement disease. SCA4 generally begins when a person is 40 or 50,…
In Wickenburg, Arizona, the community sticks together. Community means strength. So when Noah Ahnlund was diagnosed with a rare autoimmune disease called membranoproliferative glomerulonephritis type II (MPGN II/dense deposit disease)…
The ALS space looked promising in 2022 after Relyvrio’s approval and a green light for Oalsody in 2023. Then progress stalled a bit in 2024 as a result of Relyvrio…
May is Vasculitis Awareness Month. Vasculitis refers to a group of rare and often challenging diseases characterized by blood vessel inflammation. These conditions can affect anyone, at any age, and…
In the United States, Orphan Drug designation is granted by the FDA to drugs or biologics designed to treat, diagnose, or prevent a rare condition affecting fewer than 200,000 people…
Acknowledgment: This patient story is sponsored by Gilead Oncology and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant,…
Do you know what your macula does? The macula, or the central portion of your retina, plays a role in your central vision; this is required for reading, facial recognition,…
Currently, there are no approved treatment options for myotonic dystrophy type 1 (DM1), a progressive neuromuscular disease. As the disease progresses, affected individuals experience muscle weakness, respiratory distress, and cardiac…
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 Murray Walz, a…
Patients Aged 80 to 90 Respond to Standard-of-Care AML Treatment | Inside Precision Medicine
The other day I was speaking with a coaching client who suffers with a chronic health condition called dystonia, which is accompanied by chronic pain. I also live with dystonia…
Written by Rachelen Varghese I am lucky to call the Jones’ my friends. Brian Jones is a practicing nephrologist in Asheville, North Carolina who met his wife, Tia, while he…
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…
Type 1 narcolepsy, formerly known as narcolepsy with cataplexy, is a chronic neurological disorder characterized by excessive daytime sleepiness and cataplexy, or the sudden loss of muscle tone that is…
Made with fresh watermelon juice, vodka, triple sec, and lemon-lime soda, a Watermelon Crush is exactly as bubbly, bright, and refreshing as you want from your ideal summer sipper. And…
On February 28th, 2024, the first-ever White House Rare Disease Forum took place. Hosted by the White House Office of Science and Technology Policy, this forum provided a platform for…
Biopharmaceutical company Inozyme Pharma ("Inozyme") has become a leader at the forefront of developing innovative therapies for rare diseases such as ENPP1 deficiency (the childhood form is known as generalized…
May 2 is Curly Hair Day—a day to celebrate the beauty of waves, coils, and curls. But for one fifth-grader in Canada, Curly Hair Day stands as a way for…
Angelman syndrome begins at birth and although symptoms vary, most children exhibit common developmental delays such as: • Infants showing an inability to support their head • Unable to pull…
In the United States, Orphan Drug designation comes with benefits and incentives such as fee waivers, tax credits, and up to seven years of market exclusivity if/when the drug is…
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.…
It has been over four years since our lives were irreparably changed by the onset of the COVID-19 pandemic. With hundreds of millions of cases globally, and millions of associated…
In 2010, K. Pattie Figueroa of the Spencer Fox Eccles School of Medicine at University of Utah was approached by a family whose child had spinocerebellar ataxia type 4 (SCA4).…
The cause of multiple sclerosis, an autoimmune disorder impacting the nervous system, isn't well understood. Genetics, infectious agents, and environmental factors seem to all play a role, but researchers aren't…
© Copyright 2024 Patient Worthy
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.
