According to a study on hindawi.com, the presence of abnormally elevated levels of serum IgG4 was correlated with a greater likelihood of patients living with autoimmune hepatitis to present with…
Diagnosing Rare Diseases Diagnosing rare diseases is historically difficult. Physicians are taught to think about horses instead of zebras because horses are abundantly more common. For instance, if you're tired…
Takeda has just announced that their application to the the FDA for their eosinophilic esophagitis (EoE) investigative therapy has received Priority Review. This therapy is called budesonide oral suspension or…
Ryan Kress was crowned Ms. Wheelchair Virginia 2020 and Camille Schrier was crowned Miss America 2020. Besides their titles, what brings these two individuals together is their medical diagnosis with…
Written by Kendra J. Bjoraker, Ph.D., L.P. Living well with a chronic condition such as Fabry can seem contradictory. Following diagnosis, you don’t have a shelf in your mind that…
In October, I sat down with Laura and Ryan Nitahara to discuss their experience with Krabbe disease, a rare genetic lysosomal storage disorder and progressive neurological condition. Prompted by their…
In late December 2020, biopharmaceutical company AbbVie announced via press release that the prescribing information for IMBRUVICA (ibrutinib) is now updated to include long-term safety and efficacy data for…
On January 10, 2021, biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") shared positive data from its Phase 3 GENEr8-1 clinical trial. The clinical trial is evaluating an investigational gene therapy…
In a recent press release from mid-January 2020, the Pulmonary Fibrosis Foundation (PFF) announced that its registry, the PFF Registry, was being sponsored by biotechnology United Therapeutics. This sponsorship…
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