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Orsini Specialty Pharmacy Selected to be the Exclusive Specialty Pharmacy Partner for VEOPOZ™ (pozelimab-bbfg), the First and Only Treatment for CHAPLE Disease

/PRNewswire/ Orsini Specialty Pharmacy ("Orsini"), a leading independent specialty pharmacy focused on rare diseases and gene therapies, announced today. ....

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What is PLE? New procedure gives hope for others with rare condition

Protein-Losing Enteropathy (PLE) is difficult to diagnose and the treatments are limited, but the team at Nemours has come up with a unique new approach that has a very grateful surfer singing their praises. ....

United States , Debbie Rabinowitz , Katelyn Sewell , Stephanie Stahl , National Interscholastic Championships , Nemours Children , National Interscholastic , Protein Losing Enteropathy , Emmy Award Winning ,

"I have my life back": Penn Medicine solves woman's medical mystery

The Penn team will be presenting her case at a medical conference Monday. They hope the surgery will be used by other patients with her condition. ....

United States , Ronni Elena , Stephanie Stahl , Max Itkin , Todd Hecht , Penn Medicine , Protein Losing Enteropathy , Emmy Award Winning , Penn Med ,

Regeneron (REGN) Announces Pozelimab BLA for Treatment of Children and Adults with Ultra-rare CHAPLE Disease Accepted for FDA Priority Review

Regeneron (REGN) Announces Pozelimab BLA for Treatment of Children and Adults with Ultra-rare CHAPLE Disease Accepted for FDA Priority Review
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Regeneron Pharmaceuticals Inc , Drug Administration , Priority Review , Biologics License Application , Protein Losing Enteropathy , Drug Designation , Fast Track ,

Hoping for surgery in the US: Four-year-old Luis from Beuel fights for his life

The fundraising campaign "Please, help Luis!" is currently running on almost all social media channels. Luis is four years old, lives with his family in Geislar and suffers from PLE. He urgently needs an operation in the USA as life-sustaining treatment. ....

Nordrhein Westfalen , United States , Andrea Dautz , Christoph Dautz , Dieter Koch , Four Year Old Luis , Protein Losing Enteropathy , Nemours Children ,