New Therapy Helps Lower
Cholesterol in Primary Hypercholesterolemia Patients
Genzyme Corp has announced the launch of Cholestagel® (colesevelam
hydrochloride), Genzyme’s first commercially available
cardiovascular product, in Europe. Cholestagel is a new,
non-absorbed, cholesterol-lowering agent aimed at
treating patients with primary hypercholesterolemia who
cannot meet their targeted cholesterol levels with
standard therapies alone. (more)
Routine Autism Screening Recommended
All children should be screened for autism at age 18
months and again at age 2 even if they show no signs of
developmental delay, according to new guidelines from
the American Academy of Pediatrics (AAP). The hope is
that universal screening during well-child pediatric
checkups will lead to earlier diagnosis of the disorder.
(more)
Patient’s and Doctor’s Guide to Medication in
Acute Porphyria
The Canadian Porphyria Foundation have released an
e-guide about medication in acute forms of Porphyria.
The guide is available to download from their website.
(more)
BioMarin Files Investigational New Drug
Application for PEG-PAL for the Treatment of PKU
BioMarin Pharmaceutical Inc. has announced that it has
filed an investigational new drug application (IND) with
the U.S. Food and Drug Administration (FDA) for PEG-PAL
(Poly Ethylene Glycol-Phenylalanine Ammonia Lyase),
formerly known as Phenylase(TM), for the treatment of
phenylketonuria (PKU). The company expects to initiate a
clinical study of PEG-PAL in PKU patients in the first
quarter of 2008.
(more)
Gene Therapy Clinical Trial Offers Hope for
Muscular Dystrophy
A gene therapy clinical trial begins this week which
could offer new hope to muscular dystrophy sufferers. A
new treatment called molecular patch therapy has been
developed at the University of Oxford that has the
potential to give young men born with Duchenne Muscular
Dystrophy (DMD) the chance to preserve their muscle
function.
(more)
Unrelated Cord Blood Helps Infants with
Metabolic Disorders
Umbilical cord
transplants from unrelated donors can benefit infants
born with life-threatening metabolic disorders such as
Hurler disease and Krabbe leukodystrophy, according to a
study led by Duke University Medical Center researchers.
(more)
