WLKY

Enzyme treatment gives hope to children with Morquio syndrome

WEBVTT CHRISTMAS IS ALL ABOUT THE THE GIFTS. BUT FOR 3 CHILDREN - SUFFERING FROM A RARE GENETIC DISORDER - THIS SEASON IS ALL ABOUT HOPE FOR A NORMAL LIFE. TODAY - THANKS TO THE UNIVERSITY OF ...
SFGate

An infusion of hope, in any language

Every Tuesday afternoon at Children's Hospital & Research Center Oakland, the outpatient infusion unit turns into a mini United Nations, with kids from all over the world - the United States, Taiwan, ...
Yahoo

What is Morquio syndrome?

NEW HAVEN, Conn. (WTNH) — Today, we are highlighting a rare genetic disorder. Morquio syndrome challenges families and scientists worldwide. New advances in research spark hope for those living with ...
News Medical

Nemours Children's Health selected to conduct first gene therapy clinical trial for Morquio A syndrome

As part of the Foundation for the National Institutes of Health (FNIH) Accelerating Medicines Partnership ® Bespoke Gene Therapy Consortium (AMP ® BGTC), Nemours Children's Health has been chosen to ...
Healio

FDA advisory panel recommends approval of Vimizim for Morquio A syndrome

Please provide your email address to receive an email when new articles are posted on . The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee recently voted 19-2 to recommend approval of a ...
Nature

Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y

Previous studies have shown that elosulfase alfa has a favorable efficacy/safety profile in Morquio A patients aged ≥5 y. This study evaluated safety and impact on urine keratan sulfate (uKS) levels ...
Medscape

FDA Clears the First Drug for Rare Morquio A Syndrome

The US Food and Drug Administration (FDA) today approved the first drug for mucopolysaccharidosis type IVA (Morquio A Syndrome), a rare, autosomal recessive lysosomal storage disease caused by a ...
fox17online

10-year-old with rare disease may be forced to move back to Iraq, stop life-enhancing treatment

KALAMAZOO, Mich. — A 10-year-old Kalamazoo girl with an extremely rare disease may soon be without the treatment she needs. Lumar Jalil, 10, has a rare disease called "morquio syndrome". It affects ...
Monthly Prescribing Reference

Phase 3 study of GALNS for the treatment of Morquio A Syndrome

BioMarin Pharmaceuticals announced that it has initiated a Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN 110), intended for the treatment of the lysosomal storage disorder ...
Boston Magazine

Service Dog Helps a Local Fourth Grader Manage Rare Disease

When 10-year-old Bella Burton first met George, her new service dog, their connection was immediate. Rachel Burton, Bella’s mother, says that when Bella visited the animals at the Service Dog Project ...
KSL

Dream comes true for 9th grader with Morquio syndrome

This archived news story is available only for your personal, non-commercial use. Information in the story may be outdated or superseded by additional information. Reading or replaying the story in ...
Boston Magazine

Service Dog Helps a Local Fourth Grader Manage Rare Disease

When 10-year-old Bella Burton first met George, her new service dog, their connection was immediate. Rachel Burton, Bella’s mother, says that when Bella visited the animals at the Service Dog Project ...