Positive KuvanTM Pivotal Phase 3
Trial Results Published in The Lancet
BioMarin Pharmaceutical Inc has announced
that final results from the Kuvan™ (sapropterin
dihydrochloride) pivotal Phase 3 clinical trial were
published in the August 11, 2007, issue of The Lancet.
The study suggests that treatment with Kuvan results in
significant reductions in blood Phe levels in some
phenylketonuria (PKU) patients.
(more)
Effect of Miglustat in Gaucher Disease Type 1
Actelion
UK Ltd has announced the publication of "Effect of
miglustat on bone disease in adult type 1 Gaucher
disease: a pooled analysis of three multinational open
label studies" (Pastores et Al) in Clinical
Therapeutics, vol 29, number 8, 2007. It reports a
reduction of bone pain in more than 80% of the GD1
patients after 2 years miglustat therapy.
(more)
Brain Defect Helps Drive Fragile X Syndrome
A newly
discovered brain defect might be a target for the
treatment of the inherited mental disorder known as
fragile X syndrome, researchers report. The discovery
provides an understanding of how the gene mutation
responsible for the condition changes the way brain
cells communicate.
(more)
New Drug Aids Muscle Function in Myasthenia Gravis
A new drug
-- oral EN101antisense -- reduced the severity of muscle
weakness in patients with myasthenia gravis, researchers
report. The small open-label study included 16 MG
patients who took daily doses of oral EN101antisense for
four days and were monitored for a month. (more)
FDA Approves New Drug to Treat Rare Disease, Acromegaly
The U.S. Food and Drug
Administration has approved Somatuline Depot (lanreotide
acetate injection) for the treatment of acromegaly, a
rare and potentially life threatening disease in adults
caused by abnormal secretion of growth hormone (GH),
commonly from a benign tumor located in the pituitary
gland located in the brain.
(more)
Natural Chemical Found in Broccoli Helps
Combat Skin Blistering Disease
Johns Hopkins scientists have found yet another reason
why you should listen to your mother when she tells you
to eat your vegetables. Sulforaphane, a chemical present
at high levels in a precursor form in broccoli and
related veggies (cauliflower, Brussels sprouts, etc.),
helps prevent the severe blistering and skin breakage
brought on by the rare and potentially fatal genetic
disease epidermolysis bullosa simplex (EBS).
(more)
Drug Combo
Helps Fight Marfan Syndrome
Treatment with the heart drug perindopril, along with a
beta blocker, may help reduce certain cardiac
complications of the hereditary disorder Marfan
syndrome, research shows. Marfan syndrome, which
principally affects connective tissues, is often
characterized by excessive bone elongation and joint
flexibility, and eye and cardiovascular system
abnormalities.
(more)
