Navigation Links
Ultragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease
Date:5/1/2012

NOVATO, Calif., May 1, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced results from a first-in-human, multi-center, Phase 1 study of UX001 in patients with hereditary inclusion body myopathy (HIBM) showing that UX001 was well-tolerated with an expected extended release profile on absorption after oral administration.  UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency. Based on the Phase 1 results, Ultragenyx plans to initiate an international, multi-center, randomized, double-blind, placebo-controlled, parallel group Phase 2 study of UX001 in HIBM patients in the second quarter of this year.    

The Phase 1 clinical trial was a multi-center, sequential dose-escalation study designed to characterize the safety, tolerability and pharmacokinetics of UX001 Sialic Acid-Extended Release (SA-ER) tablets in patients with HIBM disease. Twenty-six subjects received SA-ER tablets orally at one of five (5) dose levels in the single-dose phase and one of four (4) dose levels in the repeat-dose phase. Preliminary data showed that SA-ER was well-tolerated at the doses evaluated, with no serious adverse events reported. The adverse event profile was unremarkable and showed no pattern or dose dependent relationship. Pharmacokinetic analysis from the study showed that single doses of SA-ER are absorbed and provide significant drug levels over a 12-16 hour period. On repeated 3 times per day dosing, serum free sialic acid concentrations reached relatively steady levels over a 24 hour cycle. 

"We are encouraged by the results of this early stage clinical trial which suggest SA-ER in single and repeated oral dosing is well-tolerated and has the potential to be a treatment for patients sufferi
'/>"/>

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

Page: 1 2 3

Related medicine technology :

1. Ultragenyx Relocates Company Headquarters to Support Operational Expansion
2. Ultragenyx Granted Orphan Designations in Europe for Two Lead Product Candidates, UX001 for HIBM and UX003 for MPS 7
3. Ultragenyx Appoints Tom Kassberg as Chief Business Officer and Announces Management Team
4. Ultragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM)
5. Ultragenyx Initiates Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Muscle Wasting Disease
6. Acella Pharmaceuticals Announces a New Addition to the Commercial Team
7. Avanir Pharmaceuticals Announces Date Of Fiscal 2012 Second Quarter Financial Results And Conference Call
8. Endo Reports First Quarter Financial Results And Announces Return To Steady Supply Of OPANA® ER And Voltaren® Gel
9. Rigel Announces First Quarter 2012 Financial Results
10. National Jewish Health, the Nations Premier Respiratory Hospital, Announces Lung Cancer Screening Trial Using Blood Test and CT
11. Jazz Pharmaceuticals Announces First Quarter 2012 Financial Results Conference Call On May 8, 2012
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:11/26/2014)... Colo. , Nov. 25, 2014 /PRNewswire/ ... ) today announced that its Chief Executive ... at the following upcoming conferences.  The public ... through webcasts on the Array BioPharma website:  ... December 3, 2014Time:1:30 p.m.  Eastern Time Location: ...
(Date:11/26/2014)... Nov. 25, 2014 Immune Pharmaceuticals Inc. ... stage biopharmaceutical company that engages in the development ... of inflammatory diseases and cancer, today announced the ... of 3,450,000 units, with each unit consisting of ... value $0.0001 per share, and one warrant to ...
(Date:11/24/2014)...  CryoLife, Inc. (NYSE: CRY ), a leading ... vascular surgery, announced today that it will participate in the ... Wednesday, December 3, 2014 at The New York Palace Hotel ... A live webcast of the Company,s presentation is scheduled to ... of the company by Pat Mackin , President and ...
Breaking Medicine Technology:Immune Pharmaceuticals Inc. Announces Closing Of Public Offering Of Common Stock And Warrants 2Immune Pharmaceuticals Inc. Announces Closing Of Public Offering Of Common Stock And Warrants 3Immune Pharmaceuticals Inc. Announces Closing Of Public Offering Of Common Stock And Warrants 4Immune Pharmaceuticals Inc. Announces Closing Of Public Offering Of Common Stock And Warrants 5CryoLife to Present at 26th Annual Piper Jaffray Healthcare Conference 2CryoLife to Present at 26th Annual Piper Jaffray Healthcare Conference 3
(Date:11/26/2014)... Dr. Parsa Mohebi is giving prospective ... With Parsa Mohebi Hair Restoration’s new winter special, patients ... FUE procedure, making it that much easier to get ... Unit Extraction”—is an advanced variety of hair restoration surgery ... Transplantation (FUT). FUE, unlike FUT, does not leave a ...
(Date:11/26/2014)... 27, 2014 Recently, Locks-Magnetic.com, a Chinese ... collection of magnetic locks on the Internet. It also ... until December. , As a leading company in ... for worldwide customers to buy high quality locks. It ... it provides locks that are not only useful but ...
(Date:11/26/2014)... (PRWEB) November 27, 2014 Boxing Day ... and tradesmen would receive gifts, known as a "Christmas ... Wikipedia ) , With the coming of Boxing Day, ... Tbdress.com is smart before every business opportunity, and it ... ”, offering a site-wide promotion for its global customers. ...
(Date:11/26/2014)... (PRWEB) November 26, 2014 Pharmaceutical ... humidity, and pressure levels during manufacturing and research ... and air conditioning) systems play a key role. ... microorganisms or bio-contaminants, while humidity can affect the ... The use of HVAC systems helps to address ...
(Date:11/26/2014)... Dallas, Texas (PRWEB) November 26, 2014 ... surgeon and Distinguished Teaching Professor in the ... Center, recently discussed facial fat compartments, fat grafting, ... Surgery, The Meeting," an annual conference held by ... importance and intricacies of this underlying facial structure ...
Breaking Medicine News(10 mins):Health News:FUE Special at Parsa Mohebi Hair Restoration This Holiday Season 2Health News:Fresh Products Launched by Famous Company Lock-Magnetic.com 2Health News:Tbdress.com: Best Boxing Day Sales For Ladies Worldwide 2Health News:Cleanroom Technology Market to Hit $3,761.9 Million by 2019 - New Research Report by MarketsandMarkets 2Health News:Cleanroom Technology Market to Hit $3,761.9 Million by 2019 - New Research Report by MarketsandMarkets 3Health News:Cleanroom Technology Market to Hit $3,761.9 Million by 2019 - New Research Report by MarketsandMarkets 4Health News:Cleanroom Technology Market to Hit $3,761.9 Million by 2019 - New Research Report by MarketsandMarkets 5Health News:Dr. Rod J. Rohrich Discusses Facelifts at ASPS Annual Assembly 2Health News:Dr. Rod J. Rohrich Discusses Facelifts at ASPS Annual Assembly 3
... Socit de Pathologie Exotique , launched shortly after the ... and English as well as meeting reports. Today, ... and is recognized for its impressive collection of tropical ... students, the Bulletin appears five times per year and ...
... suggests that exempting bars from community smoking bans makes ... and that even the most comprehensive clean indoor air ... jobs. , Researchers hope the findings, based on a ... municipalities and states considering the economic and health issues ...
... Equality,s Judicial Victory Leads to Legislative ... Guardianship CHICAGO, May 18 Today, the Illinois ... adults with disabilities under guardianship without due process. ... disabilities under guardianship has been preserved. The bill, ...
... into State-of-the-Art Neonatal Intensive Care Unit WASHINGTON, May ... home at Children,s National Medical Center. The new, ... when the babies were transported from the hospital,s ... http://www.newscom.com/cgi-bin/prnh/20090518/DC18562 )"This NICU was ...
... Now Available for Use in Clinical Trials and Population Health ... vital to health and well-being as a good night,s sleep. ... the National Sleep Foundation -- suffer from chronic sleep problems ... showing correlations between poor or insufficient sleep and disease. Now ...
... resource from ACTS Retirement-Life Communities helps consumers ask the right questions ... ... West Point, PA (Vocus) May 18, 2009 -- Preparing ... many factors to consider, including present and future needs, personal preferences, ...
Cached Medicine News:Health News:Study: Smoking bans do not cause job losses in bars and restaurants 2Health News:Study: Smoking bans do not cause job losses in bars and restaurants 3Health News:Study: Smoking bans do not cause job losses in bars and restaurants 4Health News:Passage of Illinois House Bill 2290 Adds Crucial Due Process Protection Governing Sterilization of Adults With Disabilities 2Health News:Passage of Illinois House Bill 2290 Adds Crucial Due Process Protection Governing Sterilization of Adults With Disabilities 3Health News:The Most Fragile Babies in the Nation's Capital Have a New Home 2Health News:QualityMetric Offers Way to Measure the Impact of Sleep Problems on Individuals and Groups 2Health News:7 Tips for Choosing a Retirement Community 2Health News:7 Tips for Choosing a Retirement Community 3
Maltzman-Fenzl lens manipulator, V shaped tip, straight. Also available in titanium....
0.3 mm diameter hemi-ball tip....
Vaulted shank with guard....
... individually adjusted for perfect focus where it's ... paper. This, along with Keeler's wide angle ... whatever the patient's pupil size, you will ... view. Lithium-ion technology provides a longer life ...
Medicine Products: