CRISPR was first described ten years ago as a viral mechanism for defense, taken from bacteria, which could be transformed into genetic scissors and accurately alter DNA. About Casgevy CRISPR…
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…
During the ACG Annual Scientific Meeting, which took place this year from October 20 through 25, one presenter shared insights into a multicenter retrospective cohort study which explored the efficacy…
According to a story from Pharmaceutical Business Review, the UK's National Institute for Health and Care Excellence (NICE) is backing daridorexant (marketed as QUVIVIQ) as a treatment for chronic insomnia…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
As shared by MedScape, the U.S. FDA recently approved the first treatment for people living with congenital thrombotic thrombocytopenic purpura (TTP). Its name? Adzynma. In the past, people with TTP…
Patients with severe asthma with an eosinophilic phenotype who are 12 years of age or older may now receive treatment with FASENRA as an add-on maintenance treatment. Note that…
Written by Miron Hall Because CMT is a rare disease, it often receives less attention and funding than more common medical conditions. Without adequate resources, researchers struggle to develop effective…
When 38-year-old Shereen-Fay Griffin went to the local swimming pool, she was hoping for some fun and relaxation. Instead, she contracted a rare but serious infection that left her blind…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
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…
J.D. Martinez is a designated hitter and outfielder for the LA Dodgers. According to a story from The Santa Clarita Valley Signal, he also lives with asthma, which can have…
In the United States, Orphan Drug designation is granted by the U.S. FDA to therapies or biologics intended to treat, diagnose, or prevent rare conditions affecting fewer than 200,000…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
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…
Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…
In December 2019, three months after his retirement, Paul Goodfellow received a diagnosis of invasive cancer of the bladder. He received three cycles of chemotherapy and then major surgery that…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
In the year 2000, the number of Dengue cases was reported to be approximately one-half million. In October 2023 the total reached 4.2 million cases of Dengue virus. The virus…
Every three to four minutes, someone in the U.S. is diagnosed with a blood disease such as lymphoma, leukemia, or sickle cell anemia. As reported in the Pittsburgh Post-Gazette,…
The words ‘vigorous exercise’ usually bring to mind running marathons or playing pickleball. Inside Precision Medicine reports that researchers at a Sydney, Australia University tracked the daily activity of more…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Chip Wilson, billionaire founder of Lululemon Athletica, was first diagnosed with muscular dystrophy in 1987 at the age of 32. Wilson, who is now 66 years old told Global…
In 2010, Eddie Vedder (well known for his musical career in Pearl Jam) co-founded the EB Research Partnership (EBRP) alongside wife Jill and a group of parents whose children…
Give the gift of life this holiday season and consider donating your bone marrow to BeTheMatch. Each year, over 18,000 people are diagnosed with a life-threatening blood condition that could…
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