Sentences with phrase «in sma»

Because in an SMA older prices have the same impact on the result as newer ones, traders also use moving averages that give more weight to recent prices.

Accordingly, as a condition of participating in the SMA Program, you must be an IB account holder or open and fund an account with IB.

Beginning in 2005, Dr. De Vivo and the network will conduct clinical trials in SMA patients with drugs that are currently under study as well as any compounds newly discovered by Dr. Stockwell, Dr. Monani and others.

While it is unlikely that the SMA Treatment Acceleration Act of 2009 will pass in the coming weeks, we believe that this is a time to celebrate the remarkable efforts of the entire SMA community in driving forward the Federal Government's interest in SMA research.

• SRK - 015 substantially increases lean body mass in non-human primates, with a particularly notable effect on muscles with a high proportion of fast - twitch fibers, a muscle fiber type that is particularly affected in SMA.

Other SMA investigators have recognized Dr. Monani's achievement — his mouse models are now among the most widely used in SMA research.

Alpha motor neurons are located in the ventral horn of the spinal cord and are primarily affected in SMA.

These entities attempt to remedy the primary defect in SMA: low levels of SMN protein.

Scholar Rock plans to develop SRK - 015 both in combination with therapies aimed at correcting the underlying genetic defect in SMA and as monotherapy in patients with certain subtypes of SMA.

«After I got my Ph.D., I was fortunate to meet kids with SMA at an event,» says Dr. Monani, who conducts his research in the SMA Center founded at CUMC in 2002.

The application of these compounds and technology in SMA follows on the work of Christian Lorson, PhD, Assistant Professor of Veterinary Pathobiology at the University of Missouri, who observed that certain compounds could improve the stability and distribution of the abnormal SMN2 protein in vitro, and proposed that such changes could be harnessed to modulate disease severity in SMA.

SMA researcher Dr. Rashmi Kothary from Ottawa Hospital Research Institute spoke next about musculoskeletal defects in SMA.

These defects can be rescued in mouse models by restoring SMN levels in Schwann cells, demonstrating a Schwann cell - intrinsic role in SMA pathology.

He went on to show that motor neurons in both SMA mouse models and patients have defects in myelination.

RG7916 is part of PTC's joint development program in SMA with Roche and the SMA Foundation (SMAF).

U.S. Phase 1 Trial in SMA Type 2 (STRONG) The open - label, dose - comparison, multi-center Phase 1 trial — known as STRONG — is designed to evaluate the safety, optimal dosing, and proof of concept for efficacy of AVXS - 101 in two distinct age groups of patients with SMA Type 2, utilizing a one - time IT route of administration.

This consideration has led to increased interest in SMA screening for all newborns.

Dr. Mentis» publication highlights the importance of the role of synaptic functions and neurocircuits in SMA.

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.

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

Dr. Gillingwater next focused on vascularization defects observed in SMA.

The question of whether the phenotypes observed in SMA outside of the CNS are secondary to motor neuron loss, the result of immobility, or the direct result of SMN loss in these tissues organs will be discussed in this session.

For the rest of his talk, Dr. Gillingwater focused on nonneuronal cells and tissues that are affected in SMA models and patients.

The study aims to enroll 100 children with SMA, making this one of the largest research efforts in SMA history.

However, many clinicians spoke of their own observations of peripheral symptoms in SMA patients such as gastrointestinal issues and metabolic defects that are not as common in other neuromuscular diseases, suggesting a direct role of SMN in peripheral tissues.

Dr. Darras highlighted examples from multiple published studies that demonstrate multiple tissues are affected in SMA patients including muscle, vasculature, liver, pancreas, and gastrointestinal tract.

Dr. Tom Gillingwater from the University of Edinburgh began by presenting data from Dr. Adrian Krainer's lab that showed SMN restoration in the peripheral tissues is critical for long - term survival in SMA mice, demonstrating the importance of SMN outside of the CNS.

He showed evidence of decreased bone volume in SMA mouse models of various severity, as well as increased fractures and osteoclast formation.

Dr. Rubin concluded that these results point to a likelihood of significant systemic defects in SMA patients that are potentially independent of motor neuron loss.

Research that reveals what goes wrong in SMA and suggests that a mild version of the same genetic defect may protect relatives against infection, which could explain why SMA is relatively common disease.

Through these studies he has identified a new transcription factor and associated pathway that could yield new avenues for therapeutic intervention in SMA.

They are needed for identifying and evaluating potential therapeutics that may ultimately be effective in SMA patients.

Chien - Ping Ko, PhD, University of Southern California «Synaptic defects in the spinal and neuromuscular circuitry in SMA»

A new study implicates a key cellular mechanism as defective in SMA for the first time, providing a new lead for developing future interventions.

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.

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.

Even while these experiments were proceeding, Dr. Sumner wanted to study the phenomenon of HDAC inhibition in the SMA mouse.

And it is likely to remain more of a concern in SMA than in cancer, according to Dr. Sumner, since treatment starts in childhood and is likely to be much longer.

In SMA, we don't test routinely for the gene mutation before the disease manifests, because there is no treatment.

The Foundation is also committed to raising awareness and generating support for increased research efforts in SMA among the leaders of industry and government.

Based on our recent development allowing the efficient and robust conversion of human pluripotent stem cells into affected spinal motor neurons and non - affected cranial motor neurons, our objective is to deepen the mechanisms involved in the specific degeneration of spinal motor neurons in SMA as well as the mis communication of these neurons with their muscular target.

The excitement in the SMA community is palpable, she said.

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.

The defect in SMN2 gene expression in SMA patients is at the level of premRNA splicing, such that exon 7 tends to be left out of the mRNA that ultimately makes SMN protein.

This marks a welcome new stage of advancement in SMA therapeutics development — and a clear demonstration of the value of non-profit drug development activities.

Save the Date — Society for Neuroscience 2011 Monday November 14, 2011 6:30 — 9:30 p.m. Washington Convention Center, 150B Pretzels and endplates: Motor neuron pathology and the role of SMN in motor neuron development Chien - Ping Ko, PhD, University of Southern California «Synaptic defects in the spinal and neuromuscular circuitry in SMA» George Mentis, PhD, -LSB-...]

«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.

Our second major line of research aims to understand axonal pathology in SMA, an autosomal recessive neuromuscular disease.

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.

Through consortia such as the Axon Working Group, the SMA Foundation brings together leading researchers to address critical questions in SMA.