"Morquio A syndrome
is an ultra-rare and difficult condition to treat.
The management of Morquio A syndrome
has typically been supportive and symptom-based.
In July 2014, BioMarin received USD67.5 m from Regeneron Ireland, an indirect, wholly-owned subsidiary of Regeneron Pharmaceuticals, Inc., in exchange for a voucher awarded when it received approval of Vimizim for patients with the rare disease, Mucopolysaccharidosis type IVA, also known as Morquio A syndrome
Giugliani et al., "International guidelines for the management and treatment of Morquio a syndrome
," American Journal of Medical Genetics A, vol.
M2 EQUITYBITES-November 24, 2015-BioMarin Pharmaceutical receives recommendation in England for Morquio A Syndrome
patients' access to Vimizim treatment
Bubbly Ali Zaidi was born with Morquio A Syndrome
, a condition so rare it affects just 40 patients in the UK and 400 throughout the world.
BioMarin expects to double its annual sales to USD1bn (EUR764.3m) following the launch of Galns, its enzyme replacement therapy for the treatment of Morquio A syndrome
, a rare, inherited lysosomal storage disorder.
Enzyme replacement therapy in a murine model of Morquio A syndrome
. Hum Mol Genet 2008;17(6):815-824.
The plant will be occupied in a phased transition with substantial manufacturing activities being tied to results of the ongoing Phase III trial for N-acetylgalactosamine 6-sulfatase (GALNS) for the treatment of Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome
She suffers with metabolic disorder Morquio A Syndrome
, which affects only 50 patients in the UK and 1,000 worldwide.
Young patient Sultan Ali, from Moseley, who suffers growth-stunting illness Morquio A Syndrome
, has become the first child in the world to receive the new intravenous infusion enzyme treatment.