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Phenylketonuria in Medical News

BioMarin Announces Second Quarter 2009 Financial Results

...Kuvan (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), was $16.9 million for the second quarter of 2009, compared to $12.0 ...enzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA...

Patents Issued Covering Stable Tablet Formulation and Once Daily Dosing Regimen for Kuvan

...(sapropterin dihydrochloride) for the treatment of phenylketonuria (PKU). The patent for stable tablet formulation e...HPA) due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU). Kuvan is to be used in conjunction with a ...an(R)( )(sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono,...

Patient Enrollment Complete for Phase I/II Clinical Trial for GALNS for Morquio A Syndrome

...s developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated...

BioMarin to Host Second Quarter 2009 Financial Results Conference Call and Webcast on Thursday, July 30 at 5:00 p.m. ET (23:00 CET)

...I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated...

Temporary Production Interruption at Allston Landing Manufacturing Facility Has No Impact on Aldurazyme

...I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated...

Results From Phase 1 Clinical Study of PEG-PAL in PKU and Update on Phase 2 Clinical Study

...AL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). The company expects to initiate the Phase 2 clinical study in late ...enzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA...

BioMarin to Present at the William Blair Growth Stock Conference

...I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated...

BioMarin Receives 2009 Corporate Award for the Development of Kuvan

...s toward improving the lives of people living with phenylketonuria (PKU). NORD honored BioMarin for the develop...HPA) due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU). PKU is a rare genetic disorder that can ca...ochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono,...

NORD to Honor Rare Disease Pioneers

... related to severe congenital protein C deficiency BioMarin Pharmaceutical, Inc., for developing Kuvan(R), the first specific drug therapy for phenylketonuria (PKU) Biovail Corporation, for making available to the patient community Xenazine(R), the first treatment to be approved by the Food and Drug ...

ASHG hosts fourth annual DNA Day essay contest, supports genetics education efforts

...d her understanding of how certain environmental factors can influence human health and disease. She did so by describing the surprising complexity of phenylketonuria (or PKU), which is a rare, inherited single-gene disorder that affects a person's ability to properly metabolize the amino acid phenylalanine and, if ...
Phenylketonuria in Medical Technology

BioMarin Initiates Phase 1/2 Clinical Trial for GALNS for Morquio A Syndrome

...s developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated...

BioMarin Announces FDA Approval for Kuvan

...ide) Tablets, the first specific drug therapy approved for the treatment of phenylketonuria (PKU). Shipments to the distribution channel will commence tomorrow, and Bi...h hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet. The...

Positive Kuvan(TM) Pivotal Phase 3 Trial Results Published in The Lancet

...nt with Kuvan results in significant reductions in blood Phe levels in some phenylketonuria (PKU) patients. Kuvan, an investigational oral small molecule for the treat...ropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications, which is currently in Pha...

BioMarin Partner AnGes MG, Inc. Submits BLA to Japanese Ministry of Health

...zyme Corporation. Investigational product candidates include Kuvan(TM) (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of peripheral arterial disease ...
Phenylketonuria in Medical Definition

Pathology

... - Hypervitaminosis D v • d • e Metabolic pathology ( E70-90 , 270-279 ) Amino acid Aromatic ( phenylketonuria , Alkaptonuria , Ochronosis , Tyrosinemia , Albinism , Histidinemia ) - Branched chain ( Maple syrup urine disease , Propionic acidemia , M...
Phenylketonuria in Medical Dictionary

Phenylketonuria

Phenylketonuria (PKU) is an autosomal recessive genetic disorder characterized by a deficiency in the hepatic enzyme phenylalanine hydroxylase. :541 This enzyme is necessary to metabolize the amino acid phenylalanine to the... Phenylketonuria — Comprehensive overview cov...

Tyrosinemia

... also "cures" tyrosinemia as donor enzyme is normal ... tyrosinemia is described who developed metabolic and clinical changes compatible ... phenylketonuria (PKU) is an autosomal recessive genetic disorder characterized by ... Tyrosinemia is caused by the shortage (deficiency) of one of the enzymes ....

Scurvy

... Pancreatic Cancer, Pancreatitis, Paralysis, Parasitic Diseases, Parkinson's Disease, Pelvic Inflammatory Disease (PID), Peptic Ulcer, Peritonitis, phenylketonuria ... Scurvy - Definition of Scurvy at Dictionary.com a free online dictionary with ... scorbut " scurvy ," the disease characterized by swoll...

Pku

... phenylketonuria ( PKU ) is an autosomal recessive genetic diso...alanine to the... Online resource that offers phenylketonuria ( PKU ) related information, facts, news, personal...milies and health care professionals interested in phenylketonuria ( PKU ) PKU (phenylketonuria), in its "class...

Phenylalanine

...nine . ... Excessive levels of phenylalanine in the brain can cause the levels of serotonin ... Phenylalanine isn't harmful unless you have phenylketonuria (PKU) ... PKU is a rare condition in which the body cannot process phenylalanine . ... Nutritional facts and information on amino acid phenyl...

Metabolic Diseases

...ases (IMD) Clinic consists of a multidisciplinary ... Inherited Metabolic Diseases Clinic (303) 724-2338. Related Doctors. Bellus, Gary A ... phenylketonuria and Other Metabolic Diseases : Clinical, Genetic and Newborn Screening Aspects ... on the horizon for newborn screening of metabolic diseases ? ...
Phenylketonuria in Biological News

UW-Madison study reveals new options for people with PKU

... MADISON For people with the genetic condition known as phenylketonuria (PKU), diet is a constant struggle. They can eat virtually no protein, and instead get their daily dose of this key macronutrient by drinking a bitter...

New drug holds out promise of normal diet for sufferers of devastating PKU genetic disease

...dly, Margie Fischer doesn't have to imagine it; that was normal life at her family's dinner table for years. Her daughter Maggie, now 20, suffers from phenylketonuria (PKU), a genetic disease that means her body can't tolerate anything more than a low-protein diet. PKU is described by scientists as an autosomal r...

Children's Memorial Hospital of Chicago receives a grant from BioMarin to support adult PKU outreach

...ago, IL, Children's Memorial Hospital of Chicago announced today that it has received a grant from BioMarin Pharmaceutical Inc. to support its Adult phenylketonuria (PKU) Outreach Education Project. The goal of the program is to conduct outreach to PKU patients who are no longer being treated at PKU clinics to en...

Nationwide Children's Hospital involved in expanded access program for treatment of PKU

...spital announced today its involvement in an expanded access program for sapropterin dihydrochloride, or sapropterin, an investigational treatment for phenylketonuria (PKU), a rare genetic metabolic disorder that, if left unmanaged in infants and children, can result in severe mental retardation. Currently, there ar...

Birth defects: 8 million annually worldwide

...dren with genetic disorders; Establish newborn screening programs to identify babies born with devastating but treatable metabolic disorders such as phenylketonuria (PKU); Educate women and men of childbearing age about how they can work with health professionals to maximize the chances of having a healthy pregn...
Phenylketonuria in Biological Technology

Identification of Disease Causing Mutations in Phenylketonuria by Denaturing Gradient Gel Electrophoresis Using the DCode System

Luc Michiels 1 , Baudouin Franois 1, 2 , and Jef Raus 1, 2 , 1 Dr. L. Willems-Instituut and 2 Limburgs Universitair Centrum, Universitaire Campus, B-3590 Diepenbeek, Belgium Introduction Phenylketonuria (PKU) is a hereditary...

Kuvan Receives Priority Review Status From Health Canada

...rochloride) as an oral treatment for patients with phenylketonuria (PKU) from Health Canada. Priority review provide...HPA) due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU). Kuvan is to be used in conjunction with a ...uvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono,...

BioMarin to Present at the Jefferies Healthcare Conference

...I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated...

BioMarin to Present at the Goldman Sachs Healthcare Conference

...I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated...

BioMarin to Present at the Deutsche Bank Health Care Conference

...I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated...

BioMarin to Present at the Baird Growth Stock Conference

...I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated...

BioMarin Announces First Quarter 2009 Financial Results

...Kuvan (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), was $15.5 million for the first quarter of 2009, compared to $5.8 mi...enzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA...

BioMarin to Host First Quarter 2009 Financial Results Conference Call and Webcast on Thursday, April 30 at 5:00 p.m. ET (23:00 CET)

...n developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylate...

BioMarin Receives Notice of Allowance for Once Daily Dosing Patent for Kuvan

... (sapropterin dihydrochloride) in the treatment of phenylketonuria (PKU). The company expects that the patent will b...HPA) due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU). Kuvan is to be used in conjunction with a ...ochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono,...

BioMarin to Present at the 4th Annual Citi Biotech Day

...s developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylate...
Phenylketonuria in Biological Definition

Enzyme

...ximately 2,000 types present in our bodies can lead to severe or lethal illness. An example of a disease caused by an enzyme malfunction in humans is phenylketonuria (PKU). The enzyme phenylalanine hydroxylase , which usually converts the essential amino acid phenylalanine into tyrosine does not work, resulting i...

Guthrie test

...formed on blood samples taken from the heel of the baby, at or before 4 days of age. The test typically screens for the following diseases: phenylketonuria Hypothyroidism Cystic Fibrosis Galactosemia ...
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