Navigation Links
BioMarin Initiates Phase 2 Study for GALNS in Patients Under Five Years of Age With MPS IVA
Date:11/29/2011

NOVATO, Calif., Nov. 29, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the initiation of a Phase 2 study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA (MPS IVA). 

"We expect this study to build our knowledge base in enzyme replacement therapies wherein clinicians believe earlier therapy is more impactful," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin.  "Comparatively speaking, this study is starting years before a similar study for Naglazyme, which was done as a post-marketing commitment.  We will also conduct a study in non-ambulatory patients, which, along with the study in patients under five years of age, will encompass the spectrum of MPS IVA patients not included in the Phase 3 trial.  Enrollment in the Phase 3 pivotal study is on track for results in the second half of 2012, and its execution is BioMarin's highest product development priority."

The primary objective of the Phase 2, open-label, multinational clinical study is to evaluate the safety and tolerability of infusions of GALNS at a dose of 2.0 mg/kg/week over a 52-week period in ten to fifteen MPS IVA patients under five years of age.  The secondary objectives are to evaluate urinary keratan sulfate (KS) levels and growth velocity.

About BioMarinBioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint ve
'/>"/>

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2010 PR Newswire.
All rights reserved

Page: 1 2 3

Related medicine technology :

1. BioMarin Announces FDA Approval of Expanded Biologics Manufacturing Facility
2. BioMarin to Host a Research and Development Day on December 8th
3. BioMarin Announces Third Quarter 2011 Financial Results
4. BioMarin to Present at the UBS Global Life Sciences Conference
5. BioMarin to Present at the Morgan Stanley Global Healthcare Conference
6. BioMarin to Present at the Baird Health Care Conference
7. BioMarin to Present at the Wedbush Life Sciences Conference
8. BioMarin Announces Second Quarter 2011 Financial Results
9. BioMarin to Host Second Quarter 2011 Financial Results Conference Call and Webcast on Thursday, July 28 at 5:00 p.m. ET
10. BioMarin Agrees to Acquire Biologics Manufacturing Plant in Ireland from Pfizer
11. BioMarin to Present at the William Blair Growth Stock Conference
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/23/2015)... , Jan. 23, 2015   Leatt Corporation  (OTCQB: ... protective equipment for all forms of sports, including extreme ... neck brace, announced today that the UISP Lega Motociclismo ... requires all riders to wear a neck brace during ...
(Date:1/23/2015)... 23, 2015  Mallinckrodt plc (NYSE: MNK ) ... a share repurchase program. The open-ended authorization permits the ... plc ordinary shares. "Funding additional initiatives ... as we continue to pursue a range of focused ...
(Date:1/23/2015)... and BOSTON , January 23, 2015 ... discovery and development company, is delighted to announce that the ... awarded to Miles Congreve (Vice President of Chemistry), ... Malcolm Weir (Chief Executive Officer and co-founder) for ...
Breaking Medicine Technology:Leatt Announces Neck Braces Now Required For Motocross/Minicross Racing In Italy 2Leatt Announces Neck Braces Now Required For Motocross/Minicross Racing In Italy 3Mallinckrodt plc Announces Share Repurchase Program 2Mallinckrodt plc Announces Share Repurchase Program 3Mallinckrodt plc Announces Share Repurchase Program 4Heptares Scientists to Receive the Malcolm Campbell Memorial Prize for 2015 in Recognition of Their Outstanding Contribution to GPCR Drug Discovery 2Heptares Scientists to Receive the Malcolm Campbell Memorial Prize for 2015 in Recognition of Their Outstanding Contribution to GPCR Drug Discovery 3Heptares Scientists to Receive the Malcolm Campbell Memorial Prize for 2015 in Recognition of Their Outstanding Contribution to GPCR Drug Discovery 4
... HILL, N.C., March 30, 2012  The launch landscape for ... and payer influence two of the biggest changes facing ... successful new products has never been greater with the ... development costs. In this challenging new launch ...
... PHILADELPHIA, Pennsylvania, March 30, 2012 Shire plc ... biopharmaceutical company, today announced top-line results of the PREVENT2 trial, ... mesalamine in patients with a history of diverticulitis. [ ... countries worldwide including the United States, did not meet the ...
Cached Medicine Technology:Excellence Services Program Helps Guide Pharmaceutical Industry Through the New Product Launch Landscape 2Shire Announces Top-line Results of the PREVENT2 Trial 2Shire Announces Top-line Results of the PREVENT2 Trial 3Shire Announces Top-line Results of the PREVENT2 Trial 4Shire Announces Top-line Results of the PREVENT2 Trial 5
(Date:1/22/2015)... Padre Murphy's and Owner Tom Boyle in Glendale, AZ has ... (PPHA). The PPHA's passion for others' well-being and shared ... treated for obstructive sleep apnea (OSA) has propelled them into ... the valley. The most recent plans for an event will ...
(Date:1/22/2015)... 22, 2015 Blue Cross and Blue Shield ... exemplify what it means to “Live Fearless.” , ... to share their stories about how they or someone they ... others by living in the moment. By telling these types ...
(Date:1/22/2015)... 22, 2015 A new white paper ... and person-centeredness in the long-term care environment as well ... the continuum. The paper, “The Power of Person-Centeredness in ... and practices of the patient, resident and family experience ...
(Date:1/22/2015)... Carinsurancesavings.biz has released a new blog post ... insurance . , Purchasing an accidental death and dismemberment ... This type of rider provides benefits if the insured dies ... a lesser form of life insurance. , AD&D is ...
(Date:1/22/2015)... 2015 Recently, Weddingshe.com has added many trendy ... announced its highest annual revenues in the past three years. ... glamorous wedding dresses. , According to the sales manager of ... promotions for them in 2015. This point can be reflected ...
Breaking Medicine News(10 mins):Health News:Padre Murphy's Holds NFL Retired Players Association Party Star Studded Event With Bud Light and Pro Player Health Alliance Sponsoring 2Health News:Padre Murphy's Holds NFL Retired Players Association Party Star Studded Event With Bud Light and Pro Player Health Alliance Sponsoring 3Health News:Blue Cross and Blue Shield of Minnesota to Showcase “Faces of Fearless” 2Health News:The Power of Person-Centeredness in Long-Term Care Explored by The Beryl Institute 2Health News:The Power of Person-Centeredness in Long-Term Care Explored by The Beryl Institute 3Health News:Accidental Death and Dismemberment Insurance Can Be Purchased at Low Prices Online 2Health News:Weddingshe Announces A Special Offer On Its Popular Glamorous Wedding Dresses 2
... from Cardiff University in Britain and Andrew Herxheimer from ... the newer antidepressants trigger violent behavior in people who ... were twice as likely to experience a hostile event ... sources of information in the study including data presented ...
... injury patients with moderate to severe memory loss ... according to a study//. ,Researchers, who ... injury at 19 centers across the United States, ... improved among severely impaired patients who took rivastigmine ...
... a small dispensary on the ground floor of a nursing ... last hope for a steady stream// of HIV carriers waiting ... prolong their survival. ,For the likes of ... a stigma and faced with discrimination and high cost of ...
... to haunt the prestigious All India Institute of Medical ... the elections to its resident doctors association (RDA) that ... and students of AIIMS will elect eight representatives including ... the RDA. Sixteen Candidates are contesting the elections that ...
... A study conducted at Dartmouth Medical School in Hanover, New ... among old people is normal//, yet this could be one ... ,Dr. Andrew Saykin and his associates with the ... people who suffered memory loss without dementia, another 40 patients ...
... Scientists have found that Asian-American women have the top life expectancy ... the worst// , with a gap of almost two decades between ... PLoS Medicine online they have found that the life expectancy of ... high-risk urban areas, it was 68.7 years. Leading among the 50 ...
Cached Medicine News:Health News:Drug Improves Memory Loss for Traumatic Brain Injury Patients 2Health News:AIDS Sufferers Turn to British Doctor to Prolong their Survival 2Health News:AIDS Sufferers Turn to British Doctor to Prolong their Survival 3Health News:AIIMS Goes to Polls Amid Reservation Issues 2Health News:Wide Gaps in Life Expectancy in the US 2
Bravo Tubing/Filter Supplies (Ten tubing sets, ten filters)...
... The lower esophhageal sphincter ... without using a separate manometry ... Locator, LES identification accessory kit ... used to locate the LES. ...
Buffer solution is needed to calibrate pH catheters. The specific chloride ion concentration in the Medtronic buffers is required for proper function of your Medtronic pH measurement system. Buffer s...
The WaveMax is one of the most flexible stainless steel stents due to its unique wave design and has the lowest effective profile, accommodating a 6F sheath up to 7 mm....
Medicine Products: