The US Food and Drug
Administration has approved
Vimizim (elosulfase alfa) which is
the first FDA-approved treatment
for Morquio A syndrome - also
known as Mucopolysaccharidosis
Type IVA.
Morquio A syndrome is described
by the FDA as “a rare, autosomal
recessive lysosomal storage
disease caused by a deficiency in
N-acetylgalactosamine-6-sulfatase
(GALNS)”.
There are about 800 patients
with Morquio A syndrome in the
USA, with the condition leading to
problems with bone development,
growth and mobility.
Vimizim was granted priority
review, with the move underscoring
the FDA’s “commitment to making
treatments available to patients
with rare diseases,” according to a
spokesperson for the agency.The above article was sent to subscribers in Pharmacy Daily's issue from 18 Feb 14 To see the full newsletter, see the embedded issue below or CLICK HERE to download Pharmacy Daily from 18 Feb 14
MONTU Group, its subsidiary Alternaleaf Pty Ltd, and their common director Christopher Strauch are facing legal action by the Therapeutic Goods Administration (TGA) in the Federal Court of Australia for alleged unlawful advertising on the effectiveness of their medicinal cannabis products.
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