'We are delighted to submit the Hunterase NDA, which includes data from the first enzyme replacement therapy
trial in Chinese patients to the NMPA, so soon after having licensed the treatment in January,' said James Xue, Ph.D., Founder, Chairman and CEO, CANbridge Pharmaceuticals Inc.
The enzyme replacement therapy
is designed to slow down the progression of the disease, and is being offered to five children through a "compassionate study" at London's Great Ormond Street Hospital.
KEY WORDS: Gaucher disease, Enzyme replacement therapy
(26) Babies born with the infantile-onset form of Pompe disease typically die within the first year of life, though enzyme replacement therapy
can now prolong that lifespan.
Use of enzyme replacement therapy
for Gaucher disease during pregnancy.
The court ordered that enzyme replacement therapy
be provided to a rickshaw- puller's seven- year- old son suffering from the genetic disorder.
In addition to enzyme replacement therapy
, supportive care is critical if patients with Pompe's disease are to maximize their abilities and quality of life.
"Enzyme Replacement Therapy
(ERT) is the only specific treatment available for some LSDs," she said in reply to a question.
"There are two current treatments for Hurler's syndrome: costly enzyme replacement therapy
or bone marrow transplants which require a perfectly matched donor," said Dr Sharon Byers, affiliate senior lecturer within the School of Molecular and Biomedical Sciences.
The addition of microbial beta-galactosidases directly to milk at mealtime represents a potential "enzyme replacement therapy
" for primary lactase deficiency.
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.
Tim Cox, professor of medicine at the University of Cambridge and the first UK doctor to give Gaucher patients enzyme replacement therapy
, welcomes industry investment but believes pricing at present is "completely arbitrary".