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RARE's Cholesterol Drug Evkeeza Gains EC Approval for Expanded Use
Ultragenyx Pharmaceutical RARE announced that the European Commission (EC) has expanded the eligible patient population for ...
Global Mucopolysaccharidosis Treatment Market to Witness Robust Growth, Projected to Reach USD 5,014.5 Million by 2035 Growing at a CAGR of 5.9% | FMI
Rising health awareness, increasing prevalence of various forms of mucopolysaccharidosis worldwide, continuous advances in ...
Christmas joy for boy after stem cell transplant
A five year-old boy with a life-limiting metabolic disorder is celebrating Christmas, after a vital stem cell transplant. In ...
JCR Pharmaceuticals Gains Orphan Drug Status in Japan
JCR Pharmaceuticals Co., Ltd. (JP:4552) has released an update.Don't Miss Our Christmas Offers:Discover the latest stocks recommended by top ...
BBC16d
Christmas joy for boy after stem cell transplant
In 2021, Gunner from Highley, Shropshire, was diagnosed with Mucopolysaccharidosis Type I Hurler Syndrome, which stops his body breaking down sugar. He was given one-to-two years to live without a ...