Navigation Links
BioMarin Announces Initiation of Clinical Assessment Program for Morquio A Syndrome
Date:11/3/2008

NOVATO, Calif, Nov. 3 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today the initiation of the Morquio Clinical Assessment Program (MorCAP) for patients with the lysosomal storage disease Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. MorCAP is designed to augment available data on the disease by measuring endurance and respiratory function and other parameters in affected patients. BioMarin expects to follow the MorCAP program with a Phase 1b clinical trial of enzyme replacement therapy beginning in the first quarter of 2009. The primary objectives of the Phase 1b study will be to evaluate safety and to establish the optimal dose of enzyme based on pharmacokinetic and pharmacodynamic parameters.

"After successfully advancing two enzyme replacement therapies in approximately five years each from IND filing to FDA approval, we plan to leverage our clinical, manufacturing and regulatory expertise to develop a treatment for Morquio A syndrome," said Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin. "Preliminary studies are promising and indicate that our drug candidate binds naturally to bone matrix and can adequately reach the growth cartilage after IV infusion. We recently have also shown that GALNS can reduce keratan sulfate storage in Morquio chondrocytes. This is important as the skeletal system is a primary concern in the treatment of this disease."

"We are excited to be the first site to enroll patients in the BioMarin Clinical Assessment Program for MPS IVA patients. This study is crucial to developing a deeper understanding of the clinical outcomes for this rare disorder, which will help lead to better disease management and therapy options, said Barbara B
'/>"/>

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2008 PR Newswire.
All rights reserved

Page: 1 2 3 4

Related medicine news :

1. BioMarin to Present at the Bear Stearns 20th Annual Healthcare Conference
2. BioMarin Provides Grant to NORD to Expand PKU Patient Support Services
3. BioMarin Re-Acquires Rights to Kuvan in Canada From Merck Serono
4. Fairview Specialty Pharmacy Signs Contract With BioMarin for Distribution of Kuvan(TM)
5. BioMarin to Host Fourth Quarter and Full Year 2007 Financial Results Conference Call and Webcast on Tuesday, February 26 at 5:00 p.m. ET (23:00 CET)
6. BioMarin to Present at the Cowen and Company 28th Annual Healthcare Conference
7. BioMarin to Present at the Lehman Brothers Global Healthcare Conference
8. HHS Announces $75 Million in Supplemental Funding to States for Pandemic Flu Preparedness
9. CRC Health Group Announces Acquisition of Bayside Marin Recovery Center, San Rafael
10. UHW Announces: Antelope Valley Hospital Caregivers and Board Vote to Ratify First Union Contract With SEIU UHW-West
11. HEI, Inc. Announces Sale of RFID Division Assets
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/22/2015)... (PRWEB) January 22, 2015 The City of West ... 10 a.m., to commemorate the 42nd anniversary of the Supreme Court ... , “It’s been 42 years since the Roe vs. Wade decision ... still as present as ever,” said City of West Hollywood Councilmember ...
(Date:1/22/2015)... January 23, 2015 VogueQueen is a seasoned ... prices and many designers are thrilled to have it lead ... announces its new collection of prom dresses for the global ... drive the strategic vision and he focuses on continuing the ...
(Date:1/22/2015)... AngelWeddingDress, the premier women’s dress supplier, ... dresses. View website of AngelWeddingDress.com to find ... wedding dresses online for a bridal party. AngelWeddingDress offers ... for them. Its maternity wedding dresses are specially designed ...
(Date:1/22/2015)... (PRWEB) January 22, 2015 Thousand of ... Medical Care recalled its GranuFlo and NaturaLyte dialysis concentrates ... underway in U.S. District Court, District of Massachusetts, Bernstein ... 20th, the Court has remanded a case filed by ...
(Date:1/22/2015)... Joan Lunden, award-winning American journalist, beloved television ... 32nd Annual Miami Breast Cancer Conference planned ... (PER®) , shares that “a fast-growing, virulent” cancer in ... had not followed up her clean mammogram with an ...
Breaking Medicine News(10 mins):Health News:VogueQueen Unveils New Designs of Prom Dresses for 2015 2Health News:AngelWeddingDress Has Recently Released Its Collection Of 2015 Maternity Wedding Dresses 2Health News:Court Overseeing Federal GranuFlo Lawsuits Remands Louisiana Claim Back to State Court, Bernstein Liebhard LLP Reports 2Health News:Court Overseeing Federal GranuFlo Lawsuits Remands Louisiana Claim Back to State Court, Bernstein Liebhard LLP Reports 3Health News:Court Overseeing Federal GranuFlo Lawsuits Remands Louisiana Claim Back to State Court, Bernstein Liebhard LLP Reports 4Health News:Joan Lunden Reveals Diagnostic Approach That Led To Early Detection;To Keynote 32nd Annual Miami Breast Cancer Conference® 2Health News:Joan Lunden Reveals Diagnostic Approach That Led To Early Detection;To Keynote 32nd Annual Miami Breast Cancer Conference® 3Health News:Joan Lunden Reveals Diagnostic Approach That Led To Early Detection;To Keynote 32nd Annual Miami Breast Cancer Conference® 4Health News:Joan Lunden Reveals Diagnostic Approach That Led To Early Detection;To Keynote 32nd Annual Miami Breast Cancer Conference® 5Health News:Joan Lunden Reveals Diagnostic Approach That Led To Early Detection;To Keynote 32nd Annual Miami Breast Cancer Conference® 6
... Solutions, and Blue Shield of California Look to Promote ... 17 The State of California,s efforts to move ... shot in the arm today as The California Public ... Medco Health Solutions, Inc., and Blue Shield of California ...
... PRA ) said today that A. M. Best ... The Group,s debt rating was also upgraded to "BBB." The ... http://www.newscom.com/cgi-bin/prnh/20081024/PROASSURANCELOGO ) , ... outstanding financial performance," said W. Stancil Starnes, the President and ...
... TMC207 and the discovery of new drugs to treat ... new landmark collaboration between the Global Alliance for TB ... and Tibotec Inc., (Tibotec), a global pharmaceutical company, has ... response to the urgent need to accelerate the discovery ...
... Which Currently Dominate the Market, Will Decline by $1 ... Report from Decision Resources , , WALTHAM, Mass., ... leading research and advisory firms for pharmaceutical and healthcare ... anti-amyloid monoclonal antibodies, most notably Elan/Wyeth,s bapineuzumab and Eli ...
... As job loss, bankruptcy, and divorce skyrocket with the global ... to cope. The burden of these concerns can be agonizing, ... "7 Proven Steps: How to Defeat Stress Without Going Through ... many different kinds of stress life can throw your way. ...
... June 17 Modern technology is affecting the lives ... The unique needs of elders and their sometimes faraway ... technologies. Consider the problem of an elderly person living ... to visit the doctor or to eat a meal. ...
Cached Medicine News:Health News:California Doctors to Get Wired in State's Largest e-Prescribing Initiative 2Health News:California Doctors to Get Wired in State's Largest e-Prescribing Initiative 3Health News:California Doctors to Get Wired in State's Largest e-Prescribing Initiative 4Health News:California Doctors to Get Wired in State's Largest e-Prescribing Initiative 5Health News:California Doctors to Get Wired in State's Largest e-Prescribing Initiative 6Health News:California Doctors to Get Wired in State's Largest e-Prescribing Initiative 7Health News:A. M. Best Upgrades ProAssurance Group to 'A' (Excellent) 2Health News:Unique Collaboration Between TB Alliance and Tibotec To Accelerate Tuberculosis Drug Development 2Health News:Unique Collaboration Between TB Alliance and Tibotec To Accelerate Tuberculosis Drug Development 3Health News:Unique Collaboration Between TB Alliance and Tibotec To Accelerate Tuberculosis Drug Development 4Health News:Unique Collaboration Between TB Alliance and Tibotec To Accelerate Tuberculosis Drug Development 5Health News:Unique Collaboration Between TB Alliance and Tibotec To Accelerate Tuberculosis Drug Development 6Health News:New Therapies, Including Novel Biologic Agents from Elan/Wyeth and Eli Lilly, Will Drive Robust 11 Percent Annual Growth In the Alzheimer's Disease Market 2Health News:'7 Proven Steps: How to Defeat Stress Without Going Through the Embarrassment of Counseling': Proven Ways to Defeat Stress 2Health News:'7 Proven Steps: How to Defeat Stress Without Going Through the Embarrassment of Counseling': Proven Ways to Defeat Stress 3Health News:New Technology Assists Independent Seniors 2
(Date:1/23/2015)... 2015  A new analysis of Centers for Medicare and ... shows that 81 percent of seniors chose lower-cost preferred ... at certain pharmacies. The findings were released by ... now the foundation of Medicare Part D," said Pharmaceutical ...
(Date:1/23/2015)... WYNNEWOOD, Pa. , Jan. 23, 2015  MedScope ( ... has been nominated for a 2015 Connected World ... Personal Emergency Response System (mPERS), that can be used ... M2M technology partner.  In nominating MedScope, ...
(Date:1/23/2015)... and DUBLIN , Jan. 23, ... agreement with KRS Global Biotechnology, Inc. for the compounding, packaging ... compounded for shipment in the United States ... approved Outsourcing Facility capable of compounding naltrexone tablets in various ...
Breaking Medicine Technology:New Analysis: 81 Percent of Medicare Part D Seniors Choose "Preferred Pharmacy" Plans in 2015 2MedScope Nominated by RacoWireless for Prestigious 2015 Connected World Magazine Connected World Award 2Immune Therapeutics, Inc. Announces Agreement with KRS Global Biotechnology, Inc. 2Immune Therapeutics, Inc. Announces Agreement with KRS Global Biotechnology, Inc. 3Immune Therapeutics, Inc. Announces Agreement with KRS Global Biotechnology, Inc. 4
... 12, 2011 The TandemHeart circulatory support system has ... Medicare Administrative Contractor (MAC) serving over 1.3 million beneficiaries ... of the United States. "We are extremely ... of the TandemHeart system," said  Kraig McEwen, President and ...
... Endologix, Inc. (NASDAQ: ELGX ), developer and ... details relating to the release of its second quarter 2011 ... 2011 after the close of the market. Endologix ... at 5:00 p.m. ET / 2:00 p.m. PT to discuss ...
Cached Medicine Technology:TandemHeart Approved for Expanded Reimbursement 2Endologix to Report Second Quarter 2011 Financial Results on July 21, 2011 2
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
... reference laboratory and a worldwide leader in ... an extensive test menu of highly complex ... anatomic pathology. Owned by the University of ... half of the nation's university teaching hospitals ...
Medicine Products: