Navigation Links
Isis Pharmaceuticals Initiates Phase 1b/2a Study Of ISIS-SMNRx In Patients With Spinal Muscular Atrophy
Date:11/1/2012

CARLSBAD, Calif., Nov. 1, 2012 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced the initiation of a Phase 1b/2a study evaluating ISIS-SMNRx in children with spinal muscular atrophy (SMA).  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.

"SMA is a devastating disease that leads to the premature loss of nerve cells in the spinal cord necessary for normal muscle function.  Children with SMA generally appear normal at birth, with muscle wasting and atrophy developing as early as a few months after birth.  In the most severe form of the disease, children never sit or walk and have a significantly shortened lifespan.  Although the genetic cause of SMA is well understood, the identification of an effective drug that will halt or improve the disease process has not occurred," said Richard Finkel, M.D., chief, division of neurology, Nemours Children's Hospital, Orlando, Florida.  "ISIS-SMNRx is specifically designed to intervene in the nerve cell's RNA machinery by improving splicing efficiency.  This results in an increase in the production of a normal protein, SMN, which is deficient in children with SMA.  This promising new approach could for the first time have a profound effect in children with SMA."

The Phase 1b/2a study of ISIS-SMNRx is a multiple-dose, dose-escalation study designed to assess the safety, tolerability and pharmacokinetic profile of the drug in children with SMA between the ages of 2-15 who are medically stable.   In the ongoing Phase 1 study in children with SMA, all patients have completed dosing and ISIS-SMNRx was well tolerated as a single dose administered directly into the cerebral spinal fluid.  In the Phase 1b/2a study, children with SMA will receive either two or three d
'/>"/>

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

Page: 1 2 3 4 5

Related medicine technology :

1. Auxilium Pharmaceuticals to Announce Third Quarter 2012 Results and Conduct Conference Call on Wednesday, November 7, 2012
2. BioMed Realty Announces 93,000 Square Foot Lease Expansion And Extension With Ironwood Pharmaceuticals At 301 Binney Street
3. Arena Pharmaceuticals to Host Corporate Update and Financial Results Conference Call and Webcast on Tuesday, November 6
4. Thesan Pharmaceuticals offentliggør første runde af venturefinansieringen ledt af Novo Ventures og Novartis Venture Funds i fællesskab
5. Thesan Pharmaceuticals annuncia un finanziamento di serie A co-gestito da Novo Ventures e Novartis Venture Funds
6. Thesan Pharmaceuticals gibt Serie-A-Finanzierung unter gemeinsamer Führung von Novo Ventures und Novartis Venture Funds bekannt
7. Thesan Pharmaceuticals Announces Series A Funding Co-Led By Novo Ventures And Novartis Venture Funds
8. Valeant Pharmaceuticals Reschedules Its Third Quarter 2012 Financial Results Webcast And Conference Call To November 2, 2012 Due To Hurricane Sandy
9. Neuraltus Pharmaceuticals Announces Phase 2 Clinical Results for NP001 in Patients with Amyotrophic Lateral Sclerosis (ALS)
10. Cadence Pharmaceuticals To Host Conference Call And Webcast To Discuss Third Quarter 2012 Financial Results On November 5, 2012
11. Jazz Pharmaceuticals Announces Date to Release Third Quarter 2012 Results
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/17/2014)... SAN JOSE, Calif. , Sept. 17, 2014 ... as a laureate of The Tech Awards 2014, one ... benefit humanity and spark global change. The Tech Awards, a ... by Applied Materials, Inc., selected Gradian as one of two ... - http://photos.prnewswire.com/prnh/20140917/146680 Gradian equips hospitals ...
(Date:9/17/2014)... -- , Glenmark,s first in ... in a Phase 2a proof of concept study in ... Glenmark Pharmaceuticals today announced that its first in ... 17536 has shown positive data in a Phase 2a ... conducted on 138 patients in Europe ...
(Date:9/17/2014)... , Sept. 17, 2014  Millions of people suffer ... disc disease. Recently the FDA approved the ... disc, an innovative implant device developed by Spinal Kinetics, which ... in neck and or arm pain. Hospital for Special Surgery ... the only hospital in New York City ...
Breaking Medicine Technology:Gradian Health Systems Named A 2014 Laureate of The Tech Awards 2Gradian Health Systems Named A 2014 Laureate of The Tech Awards 3Glenmark's TRPA1 Antagonist 'GRC 17536' Shows Positive Data in a Proof of Concept Study 2Glenmark's TRPA1 Antagonist 'GRC 17536' Shows Positive Data in a Proof of Concept Study 3Cervical Disc Clinical Trial for People with Neck and Arm Pain Set to Begin at Hospital for Special Surgery 2
... 10, 2011 Cephalon, Inc. (Nasdaq: CEPH ) ... percent increase compared to net sales of $2.152 billion for ... $5.66.  Excluding amortization expense and certain other items, adjusted net ... percent increase over the previous year.  This exceeded the company,s ...
... Medicare beneficiaries are reporting problems receiving medically required ... 2011 implementation of Medicare,s "competitive" bidding program in Cleveland ... than 640,000 seniors and people living with disabilities in ... The bidding program began in nine areas on ...
Cached Medicine Technology:Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 2Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 3Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 4Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 5Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 6Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 7Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 8Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 9Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 10Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 11Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 12Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 13Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 14Cephalon Reported Record Sales, Adjusted Net Income and Cash From Operations in 2010 15Seniors in Ohio Report Problems With Access to Home Medical Equipment and Services Under Controversial Medicare Bidding System 2Seniors in Ohio Report Problems With Access to Home Medical Equipment and Services Under Controversial Medicare Bidding System 3
(Date:9/17/2014)... 2014 (HealthDay News) -- Social networking sites can help ... For people who are obese, sites like Twitter and ... support from doctors as well as peers, researchers found. ... methods is that it offers the potential to be ... when compared to traditional approaches," the study,s lead author, ...
(Date:9/17/2014)... Bioluminescence, nanoparticles, gene manipulation these sound like ... in fact, they are components of an exciting ... In preclinical animal models of metastatic prostate cancer, ... VCU Institute of Molecular Medicine and Johns Hopkins ... molecular imaging approach that could revolutionize doctors, ability ...
(Date:9/17/2014)... Dallas, TX (PRWEB) September 17, 2014 ... they were recently granted several awards for their top-notch ... regarding patient experience, orthopedic care and GYN surgery. , ... Patient Safety Excellence Award™. They have received this award ... ranked among the top 5 percent in the nation ...
(Date:9/17/2014)... N.J. (PRWEB) September 17, 2014 ... advanced delivery technologies and development solutions for drugs, ... launch of OptiPact™, an integrated service and technology ... compaction, and provided from the company’s 450,000 sq. ... describes Catalent’s ability to integrate optimal formulation, development, ...
(Date:9/17/2014)... Dennis Thompson ... News) -- Exposure in the womb to household chemicals ... of developing asthma, Columbia University researchers reported in a ... increased risk of developing asthma between age 5 and ... high levels of two phthalates (pronounced thal-ates), the researchers ...
Breaking Medicine News(10 mins):Health News:Social Networking Sites Can Help Obese Lose Weight: Study 2Health News:New non-invasive technique could revolutionize the imaging of metastatic cancer 2Health News:New non-invasive technique could revolutionize the imaging of metastatic cancer 3Health News:North Central Surgical Center Receives Several Accolades 2Health News:New OptiPact™ Technology Introduced by Catalent 2Health News:Prenatal Exposure to Chemicals in Plastics Linked to Asthma Risk in Kids 2Health News:Prenatal Exposure to Chemicals in Plastics Linked to Asthma Risk in Kids 3Health News:Prenatal Exposure to Chemicals in Plastics Linked to Asthma Risk in Kids 4
... Consumption of supplemental zinc is associated with an increased ... of data from the Health Professionals Follow-up Study. ,The ... soft tissues. However, studies of the effect of zinc ... 46,000 men followed in the health professionals study, there ...
... leprosy, are the world’s most neglected diseases along ... African trypanosomiasis)and Chagas disease, that afflict the deeply ... of the global disease burden however ironically, a ... to these diseases.// , ,In a laudable initiative, ...
... than people who speak English, scientists said on Monday, ... the brain processes language. ,Unlike English speakers, who use ... scientists at the Wellcome Trust research charity in Britain ... to interpret variations in sounds in Mandarin.// ,"We ...
... problem for doctors, but what do they do when the ... to answer in a new study published in this month’s ... years worth of hospital records in Galveston, Texas, identified several ... follow when man bites man. ,The study uncovered 40 human ...
... one to suspect health problems, including heart disease. ... are at higher risk for heart attacks. ,A sore ... notes the Academy of General Dentistry. There's also a ... patients with heart conditions by diagnosing dental problems// that ...
... turn to complementary and alternative medicine, researchers have ... associated with such use. ,In this study, ... enrolled in the HIV Cost and Services Utilization ... conventional HIV care, researchers determined demographic and clinical ...
Cached Medicine News:Health News:ICMR along with other International Research organisation with drug development for Neglected Diseases, 2Health News:,Alternative Therapy in HIV Patients 2
... water perfused manometric catheters for ... diagnostic tests. All Medtronic Water ... clear, medical grade PVC tubing. ... esophagus you will find a ...
Each solid state catheter kit below is supplied with adapter cable(s) for Polygraf ID and a Manometric Calibrator....
... Medtronic offers a family of single-use ... and convenience for you. This single-use catheter ... from the risk of infection and, because ... for patient care., ,We have water ...
... of single-use products to promote safety for ... single-use catheter reduces cross-contamination and helps protect ... and, because cleaning is eliminated, you have ... have water perfused manometric catheters for esophageal, ...
Medicine Products: