Sentences with phrase «sma clinical»

2013 Jul 8; Authors: Cano SJ, Mayhew A, Glanzman AM, Krosschell KJ, Swoboda KJ, Main M, Steffensen BF, Bérard C, Girardot F, Payan CA, Mercuri E, Mazzone E, Elsheikh B, Florence J, Hynan LS, Iannaccone ST, Nelson LL, Pandya S, Rose M, Scott C, Sadjadi R, Yore MA, Joyce C, Kissel JT, International Coordinating Committee for SMA Clinical Trials Rasch Task Force

Measuring these biomarkers in SMA clinical trials may help researchers to efficiently predict and monitor SMA progression and therapeutic efficacy.

This project is supported by the Patient Advisory Group of the International Coordinating Committee for SMA Clinical Trials which includes: Families of SMA, Fight SMA, Muscular Dystrophy Association, SMA Foundation, and other SMA advocacy groups.

The Patient Advisory Group of the International Coordinating Committee (ICC) for SMA Clinical Trials has published a family - friendly set of guidelines for care in spinal muscular atrophy (SMA)-- click here to download the guide — to complement the physician guidelines published in August (for more on the physicians» consensus statement, see The Journal of Child Neurology.)

The Columbia University SMA Clinical Research Center is conducting an 18 month trial of the effects of cardiovascular and strengthening exercise on individuals with SMA Type III.

The Patient Advisory Group of the International Coordinating Committee (ICC) for SMA Clinical Trials has published a family - friendly set of guidelines for care in spinal muscular atrophy (SMA)-- click here to download the guide — to complement the physician guidelines published in August (for more on the physicians» consensus statement, see The Journal of -LSB-...]

9 February 2015: Nestlé are targeting parents with «SMA Clinical Representatives» in Boots stores on 12 February.

The public - facing marketing strategy follows Nestlé's recruitment of a national network of SMA Clinical Representatives, whose job description gives their responsibility as securing «brand endorsement» from health professionals.

Not exact matches

Nestle recruited a network of Clinical Representatives to promote relaunched SMA products to health workers.

We have been receiving reports of Nestlé's Clinical Network Representative approaching health workers inviting them to attend events on «The ins and outs of child care» — offering information on topics such as water birth (left), a free lunch and, ah yes, «New Product developments in SMA», the infant formula brand Nestlé now markets in the UK.

Since it took over the SMA brand in 2012, Nestlé has recruited a network of Clinical Representatives, whose stated responsibility is to secure brand endorsements and sales opportunities:

Nestlé has recruited a network of Clinical Representatives, with a job description giving their main responsibility as obtaining brand endorsements for SMA products from health professionals.

The job description for Nestlé's Clinical Representatives makes it very clear their job is to get health workers to endorse SMA products: http://www.babymilkaction.org/archives/3653

As Clinical Network Representative, your role is to work on the designated territory, visiting hospitals, doctors, health visitors and community midwives to develop key clinical relationships within your local health Economies, leading to opportunities for the SMA brand and Nestlé NuClinical Network Representative, your role is to work on the designated territory, visiting hospitals, doctors, health visitors and community midwives to develop key clinical relationships within your local health Economies, leading to opportunities for the SMA brand and Nestlé Nuclinical relationships within your local health Economies, leading to opportunities for the SMA brand and Nestlé Nutrition.

When Nestlé entered the UK market with the takeover of the SMA brand in 2012, it recruited a network of well - paid Clinical Representatives (who also gain bonuses), with the stated responsibilities as follows:

As promising new therapies such as those directly targeting survivor motor neuron (SMN) are entering clinical trials for infants, children, and adults with SMA, researchers are searching for biomarkers in blood that can monitor their effectiveness.

«If you consider that this is the normally bleak clinical outlook for these patients, you can understand why the ISIS - SMNRx trial represents such a watershed moment for SMA.

«We are very pleased to see the great milestone of a disease - modifying drug treatment advancing into clinical trials in SMA patients,» said Kenneth Hobby, President of Families of SMA.

The Biomarkers for Spinal Muscular Atrophy (BforSMA) clinical trial is a current pilot study looking to identify potential biomarkers (measures) that can be used to evaluate SMA disease severity and future treatments.

CARLSBAD, Calif., June 11, 2015 — Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today provided an update on its ongoing open - label Phase 2 clinical study of ISIS - SMNRx in infants with Type I spinal muscular atrophy (SMA).

The ICC's mission is to facilitate the conduct of efficient and successful clinical trials in SMA that lead to new treatments and ultimately improve care for people living with SMA.

About the SMA Foundation Founded in 2003, the Spinal Muscular Atrophy Foundation is a nonprofit organization dedicated to accelerating progress towards a treatment for Spinal Muscular Atrophy through targeted funding of clinical research and novel drug development efforts.

SMA experts and investigators discussed the importance of collaborating with other nonprofit research and patient advocacy groups; targeted genetics research; and new clinical approaches in the pipeline.

The Registry has helped recruit participants and provide data for clinical trials and other important SMA research studies.

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 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 clinical efforts, such as the Biomarkers for SMA (BforSMA) and the PNCR Network Natural History studies.

In a study reported this month in the Journal of Clinical Investigation, researchers show that a powerful HDAC inhibitor can increase SMN2 expression, ameliorate neuromuscular abnormalities, and improve the clinical phenotype of an SMA mouse model (117:659 &mdasClinical Investigation, researchers show that a powerful HDAC inhibitor can increase SMN2 expression, ameliorate neuromuscular abnormalities, and improve the clinical phenotype of an SMA mouse model (117:659 &mdasclinical phenotype of an SMA mouse model (117:659 — 671).

International Coordinating Committee for Clinical Trials The International Coordinating Committee for Clinical Trials in SMA (ICC) is a volunteer committee composed of families and patients, clinicians, advocates, industry, and regulatory agencies from the SMA community.

Preliminary clinical trials are now underway to test several HDAC inhibitors in children who have 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.

The identification of such biomarkers may help to assess drug efficacy and shorten the duration of clinical trials of SMA therapies.

The SMA Foundation initially provided more than 120 plasma samples from SMA patients collected in the Biomarkers for SMA (BforSMA) clinical study for processing on Myriad RBM's DiscoveryMAP

The SMA Foundation initially provided more than 120 plasma samples from SMA patients collected in the Biomarkers for SMA (BforSMA) clinical study for processing on Myriad RBM's DiscoveryMAP ® 250 + biomarker discovery platform.

The SMA Foundation and RBM are continuing to analyze these results and plan to create a specific panel of biomarker assays for use in clinical trials exploring new treatments for SMA.

The SMA - MAP panel is designed to evaluate the severity of SMA and disease progression and can be used to assess drug efficacy and shorten the duration of clinical trials for SMA therapeutics.

The Registry has helped recruit participants for clinical trials and has provided data for important SMA research studies.

They noted that the present findings warrant a confirmatory clinical trial for SMA rTMS on PD, undertaken on a larger scale.

The new advisors, some of the most wellrespected individuals in their fields, were specifically sought for the wealth of experience they bring to bear on Foundation efforts to advance drug development in SMA: Paul S. Anderson, PhD, retired vice president for Chemistry at Merck, G. Stephen DeCherney, MD, MPH, president of Clinical Development Services Americas for Quintiles Transnational Corp., Corey S. Goodman, PhD, president and chief executive officer of Renovis, Inc., Jonathan S. Leff, managing director at Warburg Pincus LLC and responsible for the firm's life sciences investment effort, John H. Park, Partner at Blum Capital Partners, LP, and Wesley Thompson, PhD, professor of Neurobiology at The University of Texas at Austin.

Analysis of this data revealed a set of candidate biomarkers which were then tested against over 150 longitudinal samples from an SMA natural history study by the Pediatric Neuromuscular Clinical Research Network.

«The goal of the deCODE collaboration was to generate a final optimized compound with the required properties of an effective SMA drug, called a «Clinical Candidate».

About AVXS - 101 AVXS - 101 is a proprietary gene therapy candidate of a one ‐ time treatment for SMA Type 1 and is the only clinical ‐ stage gene therapy in development for SMA.

Founded in 2003, the SMA Foundation is a nonprofit organization dedicated to accelerating progress towards a treatment and cure for Spinal Muscular Atrophy (SMA) through targeted funding of clinical research and novel drug development efforts.

«Our goal has been to expand the study of gene therapy beyond Type 1 infants to address the urgent medical needs of children with SMA Type 2, and we look forward to understanding the potential clinical impact of AVXS - 101 in these patients who, left untreated, will never walk on their own and most will never stand without assistance.»

Since its inception in 2003, the SMA Foundation has spent more than $ 100M on basic, translational, and clinical research to accelerate progress towards finding a treatment for SMA.

And because several HDAC - blocking drugs are commonly used in other fields of medicine, including neurology, it proved relatively straightforward for physicians to take some of these drugs into clinical trials in patients with SMA.

Chicago, Ill., July 20, 2016 — AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS - 101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients.

About The SMA Foundation Founded in 2003, the Spinal Muscular Atrophy Foundation is a nonprofit organization dedicated to accelerating progress towards a treatment for Spinal Muscular Atrophy through targeted funding of clinical research and novel drug development efforts.

As director of the pediatric motor disorders research program at the University of Utah School of Medicine in Salt Lake City, she led the Project Cure SMA Investigators Network, a trial consortium that recently published the results of two placebo - controlled trials of the HDAC - blocking agent valproic acid, neither of which demonstrated much clinical advantage from the drug1, 2.

According to the well - characterized natural history of the disease by the Pediatric Neuromuscular Clinical Research Network, 100 percent of children with SMA Type 2 will never walk without support, 95 percent of children will never stand without assistance and more than 30 percent will die by 25 years of age.

RG7916 is currently under investigation in two clinical studies: SUNFISH, a trial in childhood onset (Type II / III) SMA patients, and FIREFISH, a trial in infant onset (Type I) patients.

Scholar Rock is actively working to advance SRK - 015 into clinical trials to improve muscle strength and motor function in patients with Spinal Muscular Atrophy (SMA).