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…
Acknowledgment: This story is sponsored by Kite, a Gilead Company and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
In the United States, companies must obtain approval from the U.S. Food and Drug Administration to run clinical studies and transport or distribute therapies across state lines. To do so,…
On December 5, 2023, the Rare Disease Legislative Advocates (RDLA) held an RDLA Caucus briefing. This briefing, held in collaboration with the Rare Disease Congressional Caucus, provided an update on…
Written by Dr. Donna Nicholson Content Warning: Suicide, Self-harm In the summer of 1990, I learned that your life can change in the twinkling of an eye. Mine did…
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.…
In the past, Hyqvia [immune globulin infusion 10% [human] with recombinant human hyaluronidase] was approved to treat people with primary immunodeficiencies (PIs) ages two and older. Now, following data from…
In the European Union, Orphan drug designation is granted by the European Commission (EC) to drugs or biologics that are intended to diagnose, prevent, or treat rare conditions. The definition…
Written by Melany Sanchez Hello, my name is Mélany, I am 18 years old and I am from Cuba, a very poor island in the Caribbean. I was born with…
Written by Adam Faatz Imagine not being able to breathe and spending nine days climbing the world's tallest freestanding mountain. That is my story, and that is my goal. Sixty-five…
Both a hemithyroidectomy and total thyroidectomy are surgical interventions for people with low-risk differentiated thyroid cancer. Prior studies suggest that survival rates following these surgeries are relatively equal for these…
Unfortunately, clinical studies do not always work out the way they’re expected to. Biopharmaceutical company Mirum Pharmaceuticals, Inc. (“Mirum”) learned this after the Phase 2 EMBARK trial failed to meet…
On "The Golden Bachelor," 60-year-old Faith Martin started the season with a bang. She arrived on a motorcycle (badass!) before earning the First Impression rose, which my other Bachelor Nation…
A drug cannot be transported or distributed across state lines until its marketing application has been approved/cleared, so getting this clearance is crucial for many drug developers and physicians who…
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…
In 2015, the United States approved Kanuma (sebelipase alfa) for the treatment of infants living with rare lysosomal acid lipase deficiency. However, the therapy was not approved in other countries;…
According to a story from Healio, a recent study has found that use of hydroxyurea in alignment with treatment guidelines has the potential to bring down symptoms of cerebrovascular disease…
Macrophages play a crucial role in responding to injury or infection. Thomas Tapmeier, a researcher at Monash University and Oxford University, recently discussed a study he co-authored in MedicalXpress.…
An estimated 90% of the global population is infected with Epstein-Barr virus (EBV). In some cases, this infection is asymptomatic; in others, EBV can lead to numerous health impacts, including…
Michael Strahan is well-known for being an American television personality, journalist, and former NFL Defensive End (DE) for the New York Giants. Now, the family man and proud father is…
Neurogeneticist Michael Nalls of the US National Institute on Aging recently told Science Alert that a 2022 study, published in the Journal of Alzheimer’s Disease, completely changed their discovery approach.…
Many helpful guides cover immunology which is the study of the immune system and how the body defends itself against disease. The immune system is comprised of cells, organs, small…
In 2021, approximately 37 people per every 100,000 were diagnosed with idiopathic hypersomnia (IH), a neurological disorder characterized by excessive sleepiness and excessive daytime sleepiness. This amounts to roughly…
As it stands today, there are no cures for CDKL5 deficiency disorder. Treatment is symptomatic and supportive, and includes physical, occupational, and speech therapy. CDKL5 deficiency disorder causes seizures,…
AIV001 (axitinib): a potential, non-surgical therapeutic option for people with basal cell carcinoma (BCC). AiViva Biopharma describes AIV001 as: a novel formulation of a multi-kinase inhibitor combined with AiViva’s…
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