Clemson researchers conducting breakthrough research on childhood Alzheimer’s
Experts say it’s often called childhood Alzheimer’s because the symptoms are similar and it mostly impacts the brain. “It was difficult when we got the diagnosis, you are told your child is basically ...
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Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). “The path to get a treatment to the point of a BLA filing has been long and perilous for the Sanfilippo ...
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College football fans unite to raise thousands for young Ohio State fan with rare disease
It was revealed that Clark had Sanfilippo syndrome, a rare disease that is often described as childhood Alzheimers. "In my mind, I'm sitting there thinking, like, I'm not going to watch him grow ...
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Ultragenyx Submits BLA To FDA For UX111 Gene Therapy For Sanfilippo Syndrome Type A
Sanfilippo Syndrome Type A, or MPS IIIA, is a rare, fatal neurodegenerative disease in which the body is unable to break down a complex sugar molecule called heparan sulfate. This disorder ...
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RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy
Ultragenyx Pharmaceutical RARE announced that it has submitted a biologics license application (BLA) to the FDA for its UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo ...
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Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
About UX111 UX111 is a novel in vivo gene therapy in Phase 1/2/3 development for Sanfilippo syndrome type A (MPS IIIA), a rare fatal lysosomal storage disease with no approved treatment that ...
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'We step up, no matter what it is' | Vol fans raise money for young Ohio State fan with degenerative disease
Clark Willmore was recently diagnosed with Sanfilippo syndrome, a degenerative disease that causes similar symptoms to Alzheimer's but in children. James Dean, a lifelong Vol fan, saw a post on ...