In December 2023, pharmaceutical and biotechnology company Moderna announced that results from a Phase 2/3 clinical trial were published in The New England Journal of Medicine. The study explored an mRNA-based…
January 24, 2024 is recognized as Moebius Syndrome Awareness Day, a day to spread awareness about the rare disease Moebius syndrome among the medical field and the general public. The…
For the first six months of his life, Oliver Mills seemed to be developing fairly normally. He was a happy, bubbly baby. His mother Laurel found joy in every smile,…
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.…
Omega-3 fatty acids like alpha-linlenic acid or docosahexaenoic acid are "healthy fats" that are found in foods like flaxseed, fish, soybeans, and canola oil; they may also be given via…
In December 2023, clinical-stage biopharmaceutical company Clene Nanomedicine ("Clene") shared that it had met with the U.S. Food and Drug Administration (FDA) regarding Accelerated Approval for CNM-Au8®, an experimental therapy…
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…
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