Rare Classroom: Primary Immunodeficiency
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
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
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works
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
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
Duchenne muscular dystrophy (DMD) is an inherited disease that weakens a child’s muscles and eventually spreads throughout the child’s body. DMD is caused by mutations
According to a press release from the US Food and Drug Administration (FDA), the agency has approved the medication resmetirom (marketed as Rezdiffra) as the
Medication can be an essential component of managing attention deficit hyperactivity disorder (ADHD). These medications, such as Vyvanse and Adderall, can help to: Reduce symptoms
In some cases, healthcare providers can identify Angelman syndrome in utero through a prenatal ultrasound or prenatal genetic testing. But could this open the door
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
A new global study (THAOS NCT00628745) of over 6,000 people who had been diagnosed with ATTR amyloidosis found that almost one in every four patients
Ultomiris (ravulizumab) and Soliris (eculizumab), both developed by global biopharmaceutical leader AstraZeneca, are both effective treatments for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic
When you’re a baseball fan, any chance you get to join in and really experience the game is amazing. Seven-year-old Ella McKee has always been
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
Vision loss can be challenging. If you could have a one-time gene therapy that would improve your visual acuity, would you take it? It seems
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