Sentences with phrase «treating sma»

While TSA is expensive to produce and it is not approved for clinical use, similar drugs being developed to treat cancer and other diseases may be useful for treating SMA, Dr. Sumner says.

David Magnus, PhD, director of the Stanford Center for Biomedical Ethics and professor of medicine and of biomedical ethics, said those concerns resonate beyond treating SMA patients, who represent a relatively small group of people, because competition is fierce for resources to treat patients with any number of illnesses for which new and more expensive treatments are emerging.

«Targeting the splicing process is a promising strategy for finding new medicines to treat SMA, and possibly other diseases,» said Marcus Rhoades, Ph.D. of the National Institute of General Medical Sciences, which partially supported Krainer's research.

Current development - stage programs include antisense drugs to treat SMA, ISIS - SMNRx, and myotonic dystrophy type 1, ISIS - DMPKRx.

«Our strategy to treat SMA relies on a simple, powerful antisense method that boosts SMN protein levels by fixing a genetic RNA splicing glitch.

This is the first time a novel compound specifically designed to treat SMA has reached this stage in drug development.

This is the second continuation of the collaboration to fund a series of research projects initiated in 2006 to identify and optimize compounds with the potential to treat SMA by increasing production of the survival motor neuron (SMN) protein.

Not exact matches

SMA provides guidelines for finding a professional who treats selective mutism as well a directory of professionals by state.

Additionally, by becoming a treating professional member of SMA, you can also have access to peer support from colleagues who treat SM through our active Treating Professionals Ltreating professional member of SMA, you can also have access to peer support from colleagues who treat SM through our active Treating Professionals LTreating Professionals Listserv.

Treating your child can require the care of a qualified health care professional and SMA State Coordinators may be able to provide you with local recommendations based on their own experiences and feedback they have heard from other families dealing with SM.

SMA has an International Coordinator who helps link up families, teachers and treating professionals across the world with the valuable information and resources that they need.

The SMA has a lot of helpful resources for treating professionals, including handouts and information that you can find in our online library (http://www.selectivemutism.org/online-library/), reading lists for SM and child anxiety in our bookstore (http://www.selectivemutism.org/learn/shop/), as well other opportunities to learn from professionals who treat SM through our Expert Chat sessions (http://www.selectivemutism.org/learn/expert-chat/).

SMA can not, however, specifically endorse treating providers and reminds parents to carefully qualify any professional before beginning treatment.

Also, it is reassuring that our results of improved motor unit dysfunction in SMA mice treated with RG3039 mirror the independent findings by the laboratories of Drs. Sumner and Ko.

Burgart said it's an exciting time for treating spinal muscular atrophy, or SMA, and that the drug is a «game - changer» for families.

In addition, results from a recently developed biomarker assay that was designed to measure levels of SMN protein in the cerebral spinal fluid (CSF), showed time - and dose - dependent increases in SMN protein levels in SMA children treated with ISIS - SMNRx from both single - and multiple - dose studies.

The Woodlands, Texas — May 11, 2006 — Lexicon Genetics Incorporated (Nasdaq: LEXG) announced today that it was awarded a grant from the United States Army Medical Research & Materiel Command (USAMRMC) for the identification of targets that may be important in the development of drugs to prevent or treat spinal muscular atrophy (SMA), a neurodegenerative disorder and the leading genetic cause of death in early childhood.

ISIS - SMNRx is designed to treat all types of childhood SMA by altering the splicing of a closely related gene (SMN2) that leads to the increased production of fully functional SMN protein.

Through regional networks in the U.S. like the Pediatric Neuromuscular Clinical Research (PNCR) Network and Project Cure, and international organizations such as the International Coordinating Committee (ICC) and TREAT - NMD, the SMA community has collaborated effectively on recruiting patients, establishing standards of care, standardizing outcome measures, and supporting multi-site clinical efforts, such as the Biomarkers for SMA (BforSMA) and the PNCR Network Natural History studies.

The compounds in PTC's research treat the underlying cause of the disorder and demonstrate increases in SMN levels in nervous system, muscles and other tissues in SMA models.

SPINRAZA is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.

Isis is currently in collaboration with Biogen Idec (NASDAQ: BIIB) to develop and potentially commercialize the investigational compound ISIS - SMNRx to treat all types of SMA.

Nusinersen (Spinraza) will be used to treat young children with a severe and potentially fatal illness called spinal muscular atrophy (SMA)...