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Pku in Medical News

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

NOVATO, Calif., June 9 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN ) today announced results from the Phase 1 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). The company expects to initiate the Ph...

The National PKU Alliance Expands Leadership and Names Christine Brown as Executive Director

Experienced Leader Brings 15 Years of Nonprofit Expertise to National Organization SPRINGFIELD, Va., April 7 /PRNewswire/ -- The National PKU Alliance (NPKUA), the only national organization dedicated to supporting those affected by the genetic disorder phenylketonuria (PKU), today announced ...

Drug therapy for PKU reverses heart damage

A pricy drug used to treat a rare but well-known genetic disorder may hold wider promise as a treatment for millions of Americans with potentially lethal enlarged hearts, due mainly to high blood pressure, a study from Johns Hopkins shows. The common denominator in both phenylketonuria (PKU) ...

BioMarin Provides Grant to NORD to Expand PKU Patient Support Services

Financial Assistance Intended To Help Patients with Out of Pocket Expenses for Medical Foods NOVATO, Calif., Oct. 24 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that it has provided a $250,000 grant to the National Orga...

Meta-Analysis of Blood Phe Levels and Clinical Outcomes in PKU Published

NOVATO, Calif., Sept. 27 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that a systematic literature review and meta-analysis of blood phenylalanine (Phe) levels and clinical outcomes in phenylketonuria (PKU) was published in the September/October...

BioMarin Announces Second Quarter 2009 Financial Results

...art handheld device to measure blood Phe levels in pku patients. Human studies for each of these are pla... data from study of Kuvan in institutionalized pku patients 1H10: Results from Phase I/II trial ...h is currently in development for the treatment of pku and GALNS (N-acetylgalactosamine 6-sulfatase), whi...

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

...s of market exclusivity in the E.U. About pku PKU, a genetic disorder affecting approximately...mented in the 1960s and early 1970s, virtually all pku patients under the age of 40 in developed countrie...h is currently in development for the treatment of pku and GALNS (N-acetylgalactosamine 6-sulfatase), whi...

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)

...y. Other product candidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is in clinical development for the treatment of pku and GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase 1/2 clinical development for the treatment of MPS IVA. For additional infor...

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

...y. Other product candidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is in clinical development for the treatment of pku and GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase 1/2 clinical development for the treatment of MPS IVA. For additional infor...

BioMarin to Present at the William Blair Growth Stock Conference

...andidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase I clinical development for the treatment of pku and GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase I/II clinical development for the treatment of MPS IVA. For additional info...
Pku in Medical Technology

BioMarin Initiates Phase 1 Clinical Study of PEG-PAL in PKU

NOVATO, Calif., May 20 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) today announced that the first patient has initiated treatment in the Phase 1 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria ...

BioMarin Announces FDA Approval for Kuvan

...has been shown to help control blood Phe levels in pku patients, and I am thrilled that this new therapy is now commercially available to the pku community," stated Dr. Barbara Burton, Professor o... University Feinberg School of Medicine; Director, pku Clinic at Children's Memorial Hospital; and Clinic...

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

...rim, we are pleased to be able to provide Kuvan to pku patients prior to commercial availability through ...t to 50 percent of the estimated 50,000 identified pku patients in the developed world. Kuvan has receiv...e potential to address unmet medical needs. About pku PKU, a genetic disorder affecting approximately 5...
Pku in Medical Dictionary

Pku

Phenylketonuria ( PKU ) is an autosomal recessive genetic disorder characterized by a deficiency in the enzyme hepatic phenylalanine hydroxylase. :541 This enzyme is necessary to metabolize the amino acid phenylalanine to the... Online resource that offers Phenylketonuria ( ...

Phenylketonuria

...uria (pronounced fee-nill-key-toe-NURR-ee -uh) or pku is an inherited ... March of Dimes - Phenylketonuria (PKU) NIH Consensus Panel Recommendations on pku ... pku ( phenylketonuria ) is an inherited disorder that ...

Phenylalanine

...anine is a hidden danger to anyone consuming aspartame. ... be to research pku and phenylalanine extensively. Phenylalanine can be very harmful to ...... Phenylalanine isn't harmful unless you have phenylketonuria (PKU) ... pku is a rare condition in which the body cannot process phenylalanine . ... ...
Pku in Biological News

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

Imagine being forced to say no to a child crying for more food at supper. Sadly, 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 tole...

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

Chicago, 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 treat...

Unique whey protein is promising supplement for strict PKU diet

MADISON Individuals with a rare genetic condition known as phenylketonuria, or PKU, receive a difficult-to-follow prescription. They must severely limit their consumption of protein, completely avoiding mealtime staples such as meat, cheese and even bread. Not surprisingly, for many, diet is a co...

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-tasting formula of amino acids. Yet drink it they must; deviating from this...

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

Nationwide Childrens Hospital 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 seve...

ADA releases updated position statement on functional foods

...nge juice, folate-enriched breads and foods formulated with bioactive ingredients like fish oils, plant sterol esters or lutein. Medical foods include pku formulas free of phenylalanine. Foods for special dietary uses include gluten-free and lactose-free foods. ADA's position paper reviews aspects of ...

First trimester smoking linked to oral clefts

... Blood samples were taken from the children referred for surgery and their pku test samples, routinely taken at birth, were also retrieved. Their mothers ... obtained from the mother, father (after November 1998) and child, plus the pku test sample taken at birth. DNA was extracted from the samples. Four we...
Pku in Biological Technology

BioMarin Announces Roll-Out of National PKU Registry

Conference Call and Webcast to Be Held Today at 4:30 p.m. ET (21:30 CET) NOVATO, Calif., Aug. 21 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN ) announced today the roll-out of the national PKU Demographics, Outcomes and Safety Registry (PKUDOS). The registry is open to...

Biopten (Sapropterin Dihydrochloride) Approved by Japanese Ministry of Health for the Treatment of PKU

NOVATO, Calif., July 16 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that Asubio Pharma Co., Ltd. (a subsidiary of Daiichi Sankyo), has received marketing approval from the Japanese Ministry of Health, Labour and Welfare (MHLW) for a label exten...

Kuvan Receives Priority Review Status From Health Canada

...ono and Asubio, we remain dedicated to serving the pku community and providing a therapeutic option in th...ars of market exclusivity in the E.U. About pku PKU, a genetic disorder affecting approximat...mented in the 1960s and early 1970s, virtually all pku patients under the age of 40 in developed countrie...

BioMarin to Present at the Jefferies Healthcare Conference

...andidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase I clinical development for the treatment of pku and GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase I/II clinical development for the treatment of MPS IVA. For additional info...

BioMarin to Present at the Goldman Sachs Healthcare Conference

...andidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase I clinical development for the treatment of pku and GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase I/II clinical development for the treatment of MPS IVA. For additional info...

BioMarin to Present at the Deutsche Bank Health Care Conference

...andidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase I clinical development for the treatment of pku and GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase I/II clinical development for the treatment of MPS IVA. For additional info...

BioMarin to Present at the Baird Growth Stock Conference

...andidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase I clinical development for the treatment of pku and GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase I/II clinical development for the treatment of MPS IVA. For additional info...

BioMarin Announces First Quarter 2009 Financial Results

...ps fund the advancement of several promising programs including PEG-PAL for pku and GALNS for MPS IVA. We are strategically developing earlier stage progr...e), which is currently in Phase I clinical development for the treatment of pku and GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase ...

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

... United States and ten years of market exclusivity in the E.U. About pku PKU, a genetic disorder affecting approximately 50,000 diagnosed pati...n screening efforts implemented in the 1960s and early 1970s, virtually all pku patients under the age of 40 in developed countries have been diagnosed at ...

BioMarin Announces Fourth Quarter and Full Year 2008 Financial Results

...id Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "In 2009, we look forward to the advancement of several programs including PEG-PAL for pku and GALNS for MPS IVA. We also plan to continue making investments in growth opportunities as we look for attractive in- licensing or acquisition opp...
Pku in Biological Definition

Amino acid

...oxyphenylalanine) is a drug used to treat Parkinsonism . 5-HTP (5-hydroxytryptophan) has been used to treat neurological problems associated with pku (phenylketonuria). ...
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