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

of the disease provides the greatest opportunity to reap substantial improvements in muscle strength and function, potentially resulting in a lifetime of benefit. ISIS-SMNRx targets the underlying primary cause of SMA, taking advantage of the backup gene, SMN2, present in all SMA patients," said Kathryn J. Swoboda, M.D., professor, department of neurology and director of pediatric motor disorders research program at the University of Utah, School of Medicine.  "Preliminary results from Phase 1 clinical trials reveal a favorable safety profile, and appear to indicate that a single dose of the medication at the higher doses tested to date may result in sustained benefit over many months in some children. Thus, while early, we are excited to continue to work closely with Isis in further studies of ISIS-SMNRx in SMA."

"We are pleased with the progress we are making on ISIS-SMNRx.  Although there was no placebo group, the continuing improvement for up to a year after a single dose observed in this study is encouraging, particularly when considered within the context of the dose response," said B. Lynne Parshall, chief operating officer at Isis.  "Our ongoing Phase 2 program is proceeding well. The 6 mg dose group in our Phase 2 study in infants with Type I SMA has completed dosing.  Based on the safety, pharmacokinetic and pharmacodynamic profile of ISIS-SMNRx we have observed to date, we have amended the infant study to increase the dose from 9 mg to 12 mg dose. We plan to start dosing this cohort soon.  In our Phase 1b/2a multiple-dose study in children with Type II and Type III SMA, we have completed dosing in all three dose cohorts (3 mg, 6 mg and 9 mg), and we are considering adding a 12 mg dose cohort to this study.  The FDA has expressed reservations about increasing the exposure in children with Type II and Type III SMA, and
'/>"/>

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:9/15/2014)... 2014  Patients in today,s changing health care landscape ... an outpatient setting versus a hospital. In an effort ... that patients continue to receive high quality medical care; ... new internal medicine residency program. The program,s progressive curriculum ... clinic and even virtual settings. "We ...
(Date:9/15/2014)... 15, 2014 /PRNewswire-iReach/ -- Asthma is a chronic ... ages, but most commonly starts in childhood. It ... of the airways, leading to airflow limitation and ... contact with endogenous and exogenous irritants. Exposure to ... swelling of the airways, tightening of the respiratory ...
(Date:9/15/2014)... 15, 2014  Valeant Pharmaceuticals International, Inc. (NYSE: ... owned subsidiary, Valeant Pharmaceuticals International, will redeem all of ... 6.75% Senior Notes due 2017, CUSIP Nos. 91911XAL8, U9098VAD4 ... has mailed an irrevocable notice of redemption for the ... of the irrevocable notice of redemption with respect to ...
Breaking Medicine Technology:Kaiser Permanente Hawaii Launches New Internal Medicine Residency Program 2Kaiser Permanente Hawaii Launches New Internal Medicine Residency Program 3Asthma Market to Reach 161.02 million Cases in 10MM by 2023 2Asthma Market to Reach 161.02 million Cases in 10MM by 2023 3Asthma Market to Reach 161.02 million Cases in 10MM by 2023 4Asthma Market to Reach 161.02 million Cases in 10MM by 2023 5Valeant Announces Redemption of All of the Outstanding $500.0 Million Aggregate Principal Amount of its 6.75% Senior Notes due 2017 2
... AARP Illinois Disappointed By Senate Vote Against Lower Prescription Drug ... on Tuesday night rejected an amendment to the Patient Protection ... more affordable by allowing for the safe and legal importation ... endorsed by AARP and sponsored by Senators Dorgan, Snowe, McCain, ...
... , SALT LAKE CITY, Dec. 16 ... or the "Company"), a medical device company engaged in the ... announced today that Joe Pepper, PhD, has been appointed to ... Hungerford commented, "Joe has extensive experience running small to large ...
Cached Medicine Technology:AARP Illinois Key Vote News Alert 2In Preparation for Commercialization of SVARA(TM), TechniScan Appoints Joe Pepper, PhD to Board of Directors 2In Preparation for Commercialization of SVARA(TM), TechniScan Appoints Joe Pepper, PhD to Board of Directors 3
(Date:9/15/2014)... York, New York (PRWEB) September 15, 2014 ... C.R. Bard, Inc. continue to move forward in the ... Southern District of West Virginia, Bernstein Liebhard LLP reports. ... issued a Final Order on September 10th that provides ... The Order notes that none of the affected plaintiffs ...
(Date:9/15/2014)... studies, approximately one out of every 40 individuals in the ... spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles ... Missouri have made a recent breakthrough with the development of ... models of the disease. In April, a patent was filed ... our lab is using to fight SMA is to ,repress ...
(Date:9/15/2014)... 2014 This year, Avenidas Village ... 2007. To mark the special occasion, Avenidas Village would ... village and helping it to get started seven years ... fun community for seniors. Members of Avenidas Village maintain ... in their own homes. They are able to have ...
(Date:9/15/2014)... Sept. 15, 2014 (HealthDay News) -- Births of multiple ... and death than singleton births, researchers report. The ... in vitro fertilization (IVF) could ease some of that ... have increased in tandem with births to older mothers ... from the University of New South Wales, Australia. ...
(Date:9/15/2014)... 2014 Providing additional expertise to ... LLP (“the Firm”) is proud to welcome ... with the Firm’s Litigation Support Services ... experience, Wolf will assist clients in litigation support, ... advisory matters. He will also provide guidance in ...
Breaking Medicine News(10 mins):Health News:C.R. Bard Transvaginal Mesh Lawsuits Move Forward, With Issuance of New Order in Federal Multidistrict Litigation, Bernstein Liebhard LLP Reports 2Health News:C.R. Bard Transvaginal Mesh Lawsuits Move Forward, With Issuance of New Order in Federal Multidistrict Litigation, Bernstein Liebhard LLP Reports 3Health News:Researcher develops and proves effectiveness of new drug for spinal muscular atrophy 2Health News:Avenidas Village Celebrates Anniversary 2Health News:Study Ties Multiple Births to Higher Costs, Complications 2Health News:Study Ties Multiple Births to Higher Costs, Complications 3Health News:Cherry Bekaert Expands its Litigation Support Services Team Capabilities with the Addition of Steven Wolf 2Health News:Cherry Bekaert Expands its Litigation Support Services Team Capabilities with the Addition of Steven Wolf 3
... , NEW YORK, Nov. 18 WAT,s-THE-WORD?! is the first ... products, discoveries on the web and real-life experiences from a ... boys who helped their parents conceive the brand WAT-AAH! ... mom a "cooler" and better name for water, thus WAT-AAH!. ...
... 18 Banfield, The Pet Hospital® today announced ... its medical specialist/nutritionist. With nearly 20 years experience in veterinary ... platform that will position Banfield as a leader in veterinary ... as part of the Medical Quality Advancement team at Banfield,s ...
... , WASHINGTON, Nov. 18 The Union Labor ... subsidiary of ULLICO Inc., a privately held financial and ... provide pharmacy benefit management services to labor group clients. ... union employers and their members with a comprehensive pharmacy ...
... Institutet have discovered that patients with recent-onset schizophrenia have ... findings offer hope of being able to treat schizophrenia ... The causes of schizophrenia are largely unknown, and this ... that infections caught early on in life might increase ...
... have shown that lipid peroxidation stimulates collagen production ... plays an important role in the development of ... carbon tetrachloride, cholestasis and alcohol induced liver fibrosis ... effect of green tea in dimethylnitrosamine (DMN)-induced models ...
... stall carotid artery disease , WEDNESDAY, Nov. 18 (HealthDay ... additional benefit to seniors with coronary artery disease who ... , The 18-month study of 145 Baltimore-area men and ... arterial wall thickness reductions between those who took 1,500 ...
Cached Medicine News:Health News:WAT-AAH!, The First Functional Bottled Water For Kids, Announces The Release of WAT's-THE-WORD?! 2Health News:Banfield, The Pet Hospital(R) Welcomes Board Certified Nutritionist, Medical Specialist to Practice 2Health News:Banfield, The Pet Hospital(R) Welcomes Board Certified Nutritionist, Medical Specialist to Practice 3Health News:Union Labor Life Selects Prescription Solutions To Provide Pharmacy Benefit Management Services 2Health News:Union Labor Life Selects Prescription Solutions To Provide Pharmacy Benefit Management Services 3Health News:Immune system activated in schizophrenia 2Health News:Niacin Adds No Benefit for Statin Patients: Study 2
... a national reference laboratory and a worldwide ... ARUP offers an extensive test menu of ... clinical and anatomic pathology. Owned by the ... more than half of the nation's university ...
... 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 ...
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
... ARUP Laboratories is a national reference ... laboratory research and development. ARUP offers an ... unique medical tests in clinical and anatomic ... ARUP Laboratories' clients include more than half ...
Medicine Products: