Navigation Links
Isis Reports Follow-Up Data From ISIS-SMN Rx Phase 1 Study In Children With Spinal Muscular Atrophy
Date:9/19/2013

CARLSBAD, Calif., Sept. 19, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy (SMA), show that most SMA children receiving the two highest doses of the drug (6 mg and 9 mg) continued to show improvements in muscle function tests up to 14 months after a single injection of the drug.  The Phase 1 data, including these preliminary follow-on data, will be presented at the International Congress of the World Muscle Society by Dr. Kathy Swoboda on Oct. 3, 2013.  SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality.  ISIS-SMNRx is an antisense drug designed to treat all types of SMA.

(Logo: http://photos.prnewswire.com/prnh/20130807/LA60006LOGO)

The preliminary data reported today is from a follow-up analysis of 24 children with SMA who participated in a Phase 1 single-dose, open-label study of ISIS-SMNRx.  Analysis of motor function was performed in these children nine to 14 months following a single dose of ISIS-SMNRx using the Hammersmith Functional Motor Scale-Expanded (HFMSE).  The improvements in HFSME scores were dose dependent with the largest improvements observed in children in the highest dose cohort (9 mg, mean = 5.75). Most children in the 9 mg dose cohort showed continuing improvements during follow up, with no children declining.

"SMA is a devastating disease that results in severe muscle weakness and respiratory compromise in the majority of affected patients. Treating children early in the course
'/>"/>

SOURCE Isis Pharmaceuticals, Inc.
Copyright©2012 PR Newswire.
All rights reserved

Page: 1 2 3 4 5 6

Related medicine technology :

1. Qmed, the Leading Global MedTech Industry Resource, Partners with Clarimed, to Provide the Medical Manufacturing and Healthcare Industry with FDA Reports on Adverse Events
2. Sigma-Aldrich (NASDAQ: SIAL) Reports Q1 2012 Diluted EPS of $0.96, With Diluted Adjusted EPS of $0.99. Sales Increase 5% for Q1 2012. Full Year 2012 Diluted Adjusted EPS Outlook of $3.90 to $4.05 Reaffirmed.
3. Kelyniam Global Inc. Reports Record Cranial Implant Revenues in the 1st Quarter of 2012
4. Varian Medical Systems Reports Results for Second Quarter of Fiscal Year 2012
5. Mylan Reports Q1 2012 Adjusted Diluted EPS of $0.52, an 18% Increase Over Prior Year Quarter
6. CryoLife Reports Record Quarterly Revenues in First Quarter of 2012
7. PAREXEL Reports Third Quarter Fiscal Year 2012 Financial Results
8. Luminex Corporation Reports First Quarter 2012 Results
9. Hospira Reports First-Quarter 2012 Results
10. Endo Reports First Quarter Financial Results And Announces Return To Steady Supply Of OPANA® ER And Voltaren® Gel
11. The Female Health Company Reports Second Quarter Operating Results
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/22/2015)... 2015  ResMed Inc. (NYSE: RMD ) today announced ... the quarter was $423.0 million, a 10 percent increase compared ... increase on a constant currency basis). Net income was $91.2 ... ended December 31, 2013. Diluted earnings per share for the ...
(Date:1/22/2015)... DIEGO , Jan. 22, 2015  Profil Institute for ... on diabetes and obesity, announced today a new textbook,  ... Development,  published by Springer, a leading global scientific publisher. ... analyze and illustrate techniques for use in early phase ...
(Date:1/22/2015)... Jan. 22, 2015 Research and Markets ( http://www.researchandmarkets.com/research/w25xpc/allergy ... Market by Allergen Source, Product & by End User - ... The global allergy diagnostics market is valued at ... a CAGR of 12.67%, to reach $2,230.72 million by 2019. ...
Breaking Medicine Technology:ResMed Inc. Announces Results for the Second Quarter of Fiscal Year 2015 2ResMed Inc. Announces Results for the Second Quarter of Fiscal Year 2015 3ResMed Inc. Announces Results for the Second Quarter of Fiscal Year 2015 4ResMed Inc. Announces Results for the Second Quarter of Fiscal Year 2015 5ResMed Inc. Announces Results for the Second Quarter of Fiscal Year 2015 6ResMed Inc. Announces Results for the Second Quarter of Fiscal Year 2015 7ResMed Inc. Announces Results for the Second Quarter of Fiscal Year 2015 8ResMed Inc. Announces Results for the Second Quarter of Fiscal Year 2015 9ResMed Inc. Announces Results for the Second Quarter of Fiscal Year 2015 10ResMed Inc. Announces Results for the Second Quarter of Fiscal Year 2015 11New Textbook "Translational Research Methods for Diabetes, Obesity and Cardiometabolic Drug Development" Provides Critical Review Of Current And Emerging Clinical Investigation Methods 2New Textbook "Translational Research Methods for Diabetes, Obesity and Cardiometabolic Drug Development" Provides Critical Review Of Current And Emerging Clinical Investigation Methods 3Global Allergy Diagnostics (Immunoassay Analyzer, Luminometer, Assay Kit, Consumables) Market - Forecast to 2019 2Global Allergy Diagnostics (Immunoassay Analyzer, Luminometer, Assay Kit, Consumables) Market - Forecast to 2019 3
... ZANESVILLE, Ohio , May 12 Huntington Bank ... unique funding model to finance a $25 million ... region,s largest health care providers, Zanesville -based Genesis HealthCare ... install a clinical information system enabling Genesis to establish an ...
... ,, NEW YORK , May 12 Reportlinker.com announces that a new market ... , Antifungal Drugs: Technologies and ... http://www.reportlinker.com/p0196570/Antifungal-Drugs-Technologies-and-Global-Markets.html , ... , , , ...
Cached Medicine Technology:Huntington Financing Package Supports Major Regional Medical Center Electronic Medical Records Project 2Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 2Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 3Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 4Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 5Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 6Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 7Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 8Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 9Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 10Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 11Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 12Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 13Reportlinker Adds Antifungal Drugs: Technologies and Global Markets 14
(Date:1/22/2015)... Dallas, TX (PRWEB) January 22, 2015 ... the issue of experience and person-centeredness in the long-term ... moving towards action across the continuum. The paper, “The ... the Continuum,” engages perspectives and practices of the patient, ...
(Date:1/22/2015)... January 22, 2015 Carinsurancesavings.biz has released a ... death and dismemberment insurance . , Purchasing an accidental ... be very advantageous. This type of rider provides benefits if ... can be considered a lesser form of life insurance. ...
(Date:1/22/2015)... has added many trendy Champagne wedding dresses to ... the past three years. Today, the business announces a special ... the sales manager of the company, this is on the ... point can be reflected on the discounted wedding dresses from ...
(Date:1/22/2015)... EBSCO Information Services (EBSCO) and the American ... effort to further promote visibility and adoption of the top ... AMA journals available via its subscription services, EBSCO will now ... , Long known as both a subscription agency and ...
(Date:1/22/2015)... (PRWEB) January 22, 2015 Lower-Auto-Insurance.com has released a ... kit in a car for reducing the costs of an ... will also be able to get lower prices for their vehicle ... an important role in determining policy costs. Because of this, ...
Breaking Medicine News(10 mins):Health 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 2Health News:EBSCO Expands Partnership with the American Medical Association 2Health News:EBSCO Expands Partnership with the American Medical Association 3Health News:EBSCO Expands Partnership with the American Medical Association 4Health News:Driving Safe And Other Factors That Make Auto Insurance Affordable! 2
... Quest Diagnostics,Incorporated (NYSE: DGX ), the world,s ... that its third-quarter,2008 results will be made public on ... will hold its quarterly conference call with investors,to discuss ... day., The public may access the conference call ...
... leapt to the forefront of American research universities for ... to a company that analyzes intellectual property. , The ... the prowess of their properties, raised Rice to No. ... public ranking of research universities. , "The results ...
... BALA CYNWYD, Pa., Oct. 8 Philadelphia Insurance,Companies (Nasdaq: ... Coaches" will tip,off for the American Cancer Society at ... PM., Sean Sweeney, PHLY,s Executive Vice President & ... back through fitness. More than,ten thousand Americans are struck ...
... Inc. (NYSE: WTW ) today announced the ... President, North America, effective November,3, 2008. McCormick, a ... management experience, will oversee the Company,s,North American meetings ... businesses and will be reporting to David P.,Kirchhoff, ...
... HMO, Inc. announced,today that it has teamed up with ... awareness in New York City of,stroke, the number three ... Health will provide support for the associations, campaigns,against cardiovascular ... Street Run & Heart Walk and the Power to ...
... a,non-partisan, non-profit organization offering free mental health services,to ... collaboration with,HelpPRO(R) -- a leading online therapist finder ... needed mental health professionals in,their community., Through ... Give,an Hour network offering their time and professional ...
Cached Medicine News:Health News:Quest Diagnostics to Release Third Quarter 2008 Financial Results 2Health News:Rice research gets high marks 2Health News:Philadelphia Insurance Companies Slam Dunk for Cancer 2Health News:Weight Watchers Names Steven C. McCormick as President, North America 2Health News:Weight Watchers Names Steven C. McCormick as President, North America 3Health News:Touchstone Health Partners with the American Heart Association and American Stroke Association to Fight Cardiovascular Disease Among Seniors 2Health News:HelpPRO and Give an Hour Join to Help Military Families Get Free Therapy Services 2
... (MBA) is a Microsoft Windows ... management software package. MBA is ... technology and is HIPAA compliant. ... receivable management, MBA allows its ...
... In a paperless environment, providers can code ... store directly in their PDA while away from ... For those opting to use an Encounter Form/Superbill, ... entry screens. Insurance and patient payments are ...
... The HearTwave II system is ... Microvolt T-Wave Alternans (MTWA) testing ... office-based sudden cardiac death risk ... expanded versatility as it can ...
... The G-4 goniolaser lens features 4 ... complete 360° view of the anterior chamber ... fluid design (flanged glass contact element) provides ... anterior segment. 4 mirror lens ...
Medicine Products: