The research landscape for Duchenne muscular dystrophy (DMD) has been steadily increasing. Researchers are exploring more therapies, including gene therapies, that could transform the lives of those with this rare…
We are excited to announce that 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…
In 2015, the U.S. Food and Drug Administration (FDA) approved NUCALA (mepolizumab) for the treatment of severe eosinophilic asthma in adolescents and adults ages 12 and older, and by 2022,…
In mid-January 2023, Kristi Rosa of OncLive reported that the European Medicines Agency (EMA) granted Orphan Drug designation to pimicotinib (ABSK021) for inoperable tenosynovial giant cell tumors (TGCT). Orphan Drug…
Do you have cystic fibrosis? Are you searching for a stronger sense of community? Then BreatheCon, the unique virtual event spearheaded by the Cystic Fibrosis Foundation, is perfect for you! …
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…
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