Researchers recently linked Cushing syndrome remission to the development of de novo (first occurrence) diseases, including autoimmune diseases, in various patients. But how did they reach this point? The first…
A major illness can rob some people of their strength and courage, but in other cases it motivates people to get help and, if possible, to help others. 20 years…
The importance of physician education and community support in healthcare cannot be understated. Well-educated physicians are better equipped to diagnose, treat, and manage a range of medical conditions to ensure…
People who have bipolar depression face a 50% lifetime risk of attempting suicide and a 20% risk of death from suicide. Over seven million people in the U.S. are struggling…
In a clinical trial conducted by Dr. Talal and his colleagues, the team compared hepatitis C response rates for 602 patients with opioid use disorders. The trials took place…
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.…
“You have dystonia” are three words that can be very confusing and frightening. Receiving the news that you have any chronic condition can bring out a lot of emotions. People…
RINVOQ (upadacitinib) is a Janus Kinase inhibitor that is currently approved to treat a variety of conditions: moderate to severe atopic dermatitis, active psoriatis arthritis, moderate to severe rheumatoid arthritis,…
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.…
An Investigational New Drug (IND) application is a crucial part of the drug development process. INDs are requests submitted to the U.S. Food and Drug Administration (FDA) that authorize the…
Contributed by Dravet Foundation Spain The group values the progress made but urges the continued implementation of the effectiveness of the drugs Epilepsy unexpectedly and suddenly marks the onset of…
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.…
Blood transfusions are a vital medical procedure used to address a multitude of health conditions and emergencies, such as severe anemia, sepsis, trauma, internal bleeding, hemophilia, sickle cell disease, thalassemia,…
The American Society of Gene and Cell Therapy (ASGCT) held its 27th Annual Meeting from May 7-11, 2024. During the conference, stakeholders in the field of cell therapy come together…
Have you heard of antisense oligonucleotides (ASOs)? These short, synthetic strands of nucleic acids are designed to bind specifically to the mRNA transcripts of genes, blocking the production of disease-causing…
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 Ronda Thorington, the…
The FDA's Fast Track designation is designed to speed up the journey from development to approval for therapies that tackle serious conditions and meet previously unfulfilled medical needs. To learn…
Charcot-Marie-Tooth disease (CMT), though rare, is one of the most commonly inherited neurological disorders and affects peripheral nerves outside of the brain and spinal cord. Multiple CMT subtypes exist based…
Max Moore normally loved going on family walks with his mom Mandie, dad Devin, and brother Chase (age four at the time) throughout their neighborhood. But when he was eight…
Written by Martha Harlam Until 1986 I led a wonderful life. I got to sing for my supper in musicals and opera. Now, spinocerebellar ataxia was taking control of my…
According to reporting from News-Medical.net, Janssen-Cilag International NV recently announced that it had applied for European Medicines Agency (EMA) approval of its drug guselkumab (marketed as TREMFYA) in an expansion…
As reported in Biopharma Dive, the FDA’s decision to strengthen its authority involving laboratory developed tests (LDTs) has drawn considerable objections from various healthcare groups. However, the Agency has held…
In 2006, shortly after giving birth to her second child, Kelly Knight’s life drastically changed. Debilitating headaches and rapid weight gain, especially around her neck and shoulders, left her feeling…
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…
When it comes to medical research, diversity, equity, and inclusion need to be larger parts of the conversation. As it stands now, medical research can be fiercely hierarchical, expensive, and homogenous in terms of…
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