Navigation Links
Orally Delivered Compounds, Which Selectively Modify RNA Splicing Prevent Deficits In Mouse Models Of Spinal Muscular Atrophy
Date:8/7/2014

SOUTH PLAINFIELD, N.J., Aug. 7, 2014 /PRNewswire/ -- Today the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene starting early after birth is preventing deficits in mouse models of Spinal Muscular Atrophy (SMA). Scientists from Roche Pharma Research and Early Development (pRED), PTC Therapeutics, Inc., the SMA Foundation, the University of Southern California and Harvard University collaborated to demonstrate that continuous treatment of SMA mice with these compounds increased life span, normalized body weight and prevented both disease-related motor dysfunction and neuromuscular deficits in a mouse model of SMA. To view the animation related to the study, please visit: SMA video

"The investigational compounds used in this study represent the first orally available SMN2 splicing modifiers for SMA," commented Stuart W. Peltz, CEO of PTC Therapeutics, Inc. "Using the experience and expertise in RNA biology we have gained at PTC over the last 16 years, we used our alternative splicing technology to identify and subsequently optimize investigational compounds that target the SMN2 splicing to produce the SMN protein. Our unique partnership with Roche and the SMA Foundation has allowed this project to rapidly move into clinical development."

"Although still preclinical, these results demonstrate how SMN2 splicing modifiers could correct the molecular deficit that causes SMA," said Luca Santarelli, Head of Neuroscience, Ophthalmology and Rare Diseases at Roche. "This study represents an important step towards developing a potential therapeutic option for this devastating and currently untreatable condition. Early clinical trials are currently underway to determine the safety and tolerability of this approach."

The
'/>"/>

SOURCE PTC Therapeutics, Inc.
Copyright©2014 PR Newswire.
All rights reserved

Page: 1 2 3 4 5

Related medicine technology :

1. Researchers Developing New Multiple Sclerosis Drug That Can Be Taken Orally
2. Orally Disintegrating Tablets and Thin Films Poised to See Strong Growth
3. Kinex Announces First Patient Dosed in Phase Ib Study of Oraxol, an Orally Dosed Paclitaxel
4. BioLineRx Announces New Positive Results in Phase 2a trial for Orally Available Inflammatory Bowel Disease Treatment
5. Synthetically Optimized HIF-1 Alpha DNA Delivered with Inovios Electroporation Technology Provides Significant Therapeutic Effects for Peripheral Arterial Disease in Animal Model
6. Innovative Compounds, Combination Therapies Improve Care of Patients with Wide Range of Blood Disorders
7. Inovio Initiates H1N1 Universal Influenza Vaccine Clinical Trial Targeting Over-65 Population, Which Represents 90% of Flu Deaths
8. Ranbaxy Commends New IMS Study Which Credits Generic Prescription Drugs With $1 Trillion Savings In U.S. Health Care Costs Over Past 10 Years
9. Increasing Affluence and Legislative Reforms are Transforming Polands Pharmaceutical Market, Which Holds Strategic Importance for Multinational Companies
10. West Completes Preliminary Study Evaluating the SmartDose Electronic Patch Injector Technology Platform, Which Incorporates a Daikyo Crystal Zenith Cartridge
11. Half of Surveyed Nephrologists Report Moderate Familiarity with Velphoro, Which Received FDA Approval Late Last Year for the Treatment of Hyperphosphatemia in Dialysis
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/1/2014)... Texas , Oct. 1, 2014 ... is scaling volume production of photoactive quantum dots ... technologies . While offering numerous advantages for solar ... producing large quantities of quantum dots with which ... now kept them from commercial utilization and acceptance. ...
(Date:10/1/2014)... , Oct. 1, 2014 ... a clinical-stage biopharmaceutical company focused on cardio-renal, ... results from its 371 patient Phase 2b ... constipation-predominant irritable bowel syndrome (IBS-C).  Results from ... meaningful improvement in IBS-C symptoms for tenapanor-treated ...
(Date:10/1/2014)... Switzerland , October 1, 2014 /PRNewswire/ ... Clinical Genomics and Next Generation Sequencing (NGS) data analysis, ... to run full cystic fibrosis analysis in a single ... and characterisation of all types of variants in a ... Until now, it has not been possible to ...
Breaking Medicine Technology:Quantum Materials Scaling Up Photoactive Quantum Dot Production for Solar Power Generation 2Quantum Materials Scaling Up Photoactive Quantum Dot Production for Solar Power Generation 3Ardelyx Reports Positive Results from Its Phase 2b Clinical Trial Evaluating Tenapanor in IBS-C Patients 2Ardelyx Reports Positive Results from Its Phase 2b Clinical Trial Evaluating Tenapanor in IBS-C Patients 3Ardelyx Reports Positive Results from Its Phase 2b Clinical Trial Evaluating Tenapanor in IBS-C Patients 4Ardelyx Reports Positive Results from Its Phase 2b Clinical Trial Evaluating Tenapanor in IBS-C Patients 5Ardelyx Reports Positive Results from Its Phase 2b Clinical Trial Evaluating Tenapanor in IBS-C Patients 6Sophia Genetics Launches Full CFTR Analysis in Single Clinical Grade NGS Experiment 2
(Date:10/2/2014)... HealthDay Reporter WEDNESDAY, Oct. 1, 2014 ... raise your risk for high blood pressure, a new study ... lived within 109 yards of a busy road had a ... women living at least half a mile away, researchers report. ... of our physical environment on our health and well-being," said ...
(Date:10/2/2014)... HealthDay Reporter WEDNESDAY, Oct. 1, 2014 (HealthDay News) -- ... may face an increased risk of dying in the next several ... older Americans, researchers found those who were unable to detect scents ... as likely to die in the next five years, versus those ... the inability to distinguish odors -- was a bigger predictor of ...
(Date:10/2/2014)... HealthDay Reporter WEDNESDAY, Oct. 1, 2014 (HealthDay ... have a total of 500 confirmed cases of Enterovirus D68, ... since the summer, U.S. health officials reported Wednesday. Four ... but it,s not clear what role -- if any -- ... officials are also trying to determine if the virus is ...
(Date:10/1/2014)... University of Adelaide have developed a model that could ... outcome from treatment - from their very first psychotic ... of factors, including clinical symptoms, cognitive abilities, MRI scans ... blood. , Speaking in the lead up to World ... Psychiatry , Professor Bernhard Baune , says ...
(Date:10/1/2014)... shown that more than 80 per cent of bowel ... study found that medicines called ,JAK inhibitors, halted tumour ... is present in more than 80 per cent of ... are in clinical trials, for diseases including rheumatoid arthritis, ... is the second-most common cancer in Australia with nearly ...
Breaking Medicine News(10 mins):Health News:Living Near a Highway May Be Bad for Your Blood Pressure 2Health News:Living Near a Highway May Be Bad for Your Blood Pressure 3Health News:Could a Fading Sense of Smell Point to Earlier Death? 2Health News:Could a Fading Sense of Smell Point to Earlier Death? 3Health News:42 States Reporting Respiratory Virus That Targets Kids 2Health News:42 States Reporting Respiratory Virus That Targets Kids 3Health News:Predicting the future course of psychotic illness 2Health News:Eighty percent of bowel cancers halted with existing medicines 2
... citrus fruits rich in Vitamin C such as papaya, oranges ... research has found that the nutrient could help reduce ... ,Researchers from the University of Warwick have found that the ... in diabetics. ,Free radicals are tissue-damaging molecules, and ...
... and smoke-free work environment play key roles in helping people ... a study has found. ,The study was ... environmental factors are more influential than individual behaviours and beliefs ... researcher Jon Macy, said that though smoking cessation attempts often ...
... , ... ... ... clutches? Even fetuses in wombs experience stress! Stress can stem from a variety of causes-relationships, job, school and daily activities-just about anything. ...
... Working in an environment where smoking is allowed is especially ... that is not considered safe at any level, a ... by researchers at the Multnomah County Health Department and Oregon ... elevated levels of the carcinogen NNK, which is found in ...
... led by a Children's Hospital of Eastern Ontario (CHEO )researcher ... to vitamin D deficiency Rickets. Despite the fact that generation ... supplements and sunshine, vitamin D deficiency rickets has persisted in ... disease that weakens the leg bones and stunts growth, the ...
... death rates for men from the rural areas are 10 ... ,Despite The fact that cancer survival rates are improving overall, ... death rate of men living in rural areas and their ... in Australia has risen from 78,857 new cases in 1996 ...
Cached Medicine News:Health News:Smoke-free Workplace, Non-smoking Spouse Help Quitters Stay Off the Butt 2Health News:Living With Stress 2Health News:Living With Stress 3Health News:Living With Stress 4Health News:Secondhand Workplace Smoke Hits Non-smokers Badly 2Health News:Vitamin D Deficiency Still Persists in Babies 2Health News:Cancer Deaths of Rural Men on the High 2
... The Repeater Pipette* provides quick, convenient, ... procedures. Eppendorf Combitips have a polyethylene ... them ideal for use with most ... Product features: Five separate volumes can ...
... The only catheter of its kind, ... rapidly collects real-time cardiac electrical information, ... map. With 64 electrodes, this unique ... more than 3,000 points of electrical ...
... St. Jude Medical's universal programmer platform, ... used in combination with St. Jude ... products for emerging indications. Features ... touch-sensitive, active matrix LCD screen Programmer ...
... Coronary Stent System introduces ... lesion access and conformability. ... low crossing profile, and ... the performance of the ...
Medicine Products: