One of the most successful healthcare services benefiting the public is newborn screening. Unfortunately, this life-saving health initiative is not provided for all rare diseases in some U.S. states. As…
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…
In a late July 2023 news release, global healthcare company Grifols shared that positive topline data was now available from a Phase 4 clinical study evaluating XEMBIFY (immune globulin…
A primary immunodeficiency (PI), or primary immune deficiency disease, is an inherited immune system disorder caused by gene mutations which prevent the immune system from functioning properly. It can…
Near the start of August 2022, biotechnology company Inmagene Biopharmaceuticals ("Inmagene") and biopharmaceutical company HUTCHMED shared via news release that the first participant had been dosed in a Phase…
We’ve reached the month of May, and you know what that means – it is Guillain-Barre syndrome (GBS) and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Awareness Month! During this month, the…
It is extremely important to get the right medications into the hands of patients who need them. Recently, the FDA helped do this for pediatric patients (aged 2+) with primary…
The National Organization for Rare Disorders (NORD) held its virtual Rare Diseases and Orphan Products Breakthrough Summit on October 18-19, 2021. Patient Worthy was able to sit in on some…
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