In the United States, a rare condition is defined as one affecting fewer than 200,000 people nationwide. But when you consider how many people actually live with a rare condition,…
What’s better than being in warm and sunny Arizona? Getting to learn alongside the amazing primary biliary cholangitis (PBC) community while there! The PBCers organization held its 2024 Patient Education…
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…
These efforts led to collaboration by the International Brain pH Project that involves 131 scientists in 105 labs from seven countries. The scientists have identified changes in lactate levels…
Anna-Lena Neehus of the Imagine Institute at Paris Cité University was searching through genomic data from over 15,000 patients. She wanted to understand how and whether genetic deficiencies contributed to…
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…
Perhaps 70- 85% of rare diseases have a root in genetics, - which is why continuous research and reporting is so critical for those with rare conditions. This is exactly…
Neurological conditions now have the highest rates of disability and ill health worldwide. The Disparity Although more than 80% of the neurological deaths and nervous system disorders occur in lower…
A study by a team of scientists at the Children’s Research Institute was reported this week in the journal Nature Communications. The study is welcomed by the medical profession as…
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…
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.…
This opinion piece was provided by Kevin Woodward of Phoenix, Maryland. Kevin is a member of the Barth Syndrome Foundation board of directors. The role of the U.S. Food and…
On March 7, 2024, the United Kingdom’s Medicines and Healthcare products Regulatory Agency shared via press release that the agency has approved Ztalmy (ganaxolone) for people living with CDKL5 deficiency…
Prader-Willi syndrome (PWS) is a rare genetic disorder with a number of physical, emotional, mental, and behavioral manifestations. Individuals with PWS may exhibit cognitive impairment, hypotonia (poor muscle tone), and…
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…
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.…
, organized by the EveryLife Foundation for Rare Diseases, is one of the most important annual advocacy events for the rare disease patient community. The event is held the last…
According to a story from BioSpace, the current standard approaches for drug development aren't well suited for rare diseases, which have unique characteristics in comparison to more widespread illnesses. As…
Contributed by Scott Gray. Scott Gray is the co-founder and CEO of Clincierge, a provider of patient support services for clinical trials. Since 2015, Clincierge patient coordinators have managed logistics and reimbursements in…
Introduction I am a 50-year-old woman from the UK who has been suffering with an undiagnosed condition for 11-years for which I am seeking a diagnosis and medical treatment. I…
Having a rare disease can be challenging and isolating. It's often difficult to describe the experience to someone who hasn't gone through it. Mobilizing funds for support, resources, and research…
Out of 103,000 people in the United States waiting for a transplant, almost 88,000 individuals on the waiting list need a kidney transplant. These statistics were recently provided through Organ…
When it comes to rare disease, rare isn't actually that rare. More than 10,000 rare diseases have been identified. Rare diseases affect more than 350 million people across the globe…
Quinnlyn Walters is a bright, bubbly, and active 8-year-old. She loves reading, riding horses, swimming and softball, karate, Star Wars, and spending time outside with her friends and family. But…
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