Sentences with phrase «studying sma»
«We have many questions about the disease that we need to use mouse models to answer,» says Dr. Monani, who came to CUMC about a year ago and has been studying SMA for a decade.
http://www.smafoundation.org/
Researchers who are interested in studying SMA can request two types of data from the Registry, de-identified information and identifiable information.
The Registry connects patients and families interested in participating in research and researchers interested in studying SMA.
Not exact matches
The study, released today, finds that using SMAs costs 23 percent less than employing a direct sales force at retail.
Commissioned jointly by the Grocery Manufacturers Association (GMA) Sales Agency Committee and the Association of Sales and Marketing Companies (ASMC) Foundation and conducted by Bain & Company, the study provides an analysis of performance results of CPG companies before and after outsourcing to SMAs.
Section 7: Advertising [the health worker is instructed to open an envelope containing a Nestlé advertisement for the SMA HA milk it is currently promoting in journals and at the study days]
She is the Director of Membership at SMA, the Washington State Coordinator, and is also studying to receive her BCBA.
10 November 2015: Nestlé is targeting health workers in the UK again with SMA branded study days.
«SMA Study Days are designed to provide educational support to HCPs working in infant and maternity care.
According to studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time.
«Encouraging data for gene replacement therapy for SMA type I, phase 1 study shows.»
According to recent studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time.
Lorson's study, «Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models,» was published in September 2014 in the Journal of Human Molecular Genetics.
The study took advantage of an important large animal model of SMA, the SMA piglet developed by the research group.
«Upsetting a fragile alliance triggers a deadly childhood disease: Study bolsters the fight against SMA, the leading genetic killer of newborns.»
«Another intriguing aspect of our study is the molecular link we have established between ALS and spinal muscular atrophy or SMA, which is a pediatric motor neuron disease,» says Edens.
Using sophisticated technology, the study found that mitochondria in SMA motor neurons produce energy at a slower rate, depleting the nerve.
This time the study concerned SMA type 2, in which the illness occurs when children have learned to sit by themselves but have not yet begun to walk.
But the study also found that before 2008, only 36 % of vessel - strike deaths occurred in zones that would become SMAs.
NEW YORK, NY — October 24, 2008 — The Spinal Muscular Atrophy (SMA) Foundation, a nonprofit organization dedicated to accelerating the development of a treatment for SMA, is pleased to announce the launch of a pilot study of Biomarkers for Spinal Muscular Atrophy (BforSMA).
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.
In the interim analysis of this ongoing multiple - dose Phase 1b / 2a study, children with Type II or Type III SMA were dosed intrathecally with 3 mg, 6 mg or 9 mg of ISIS - SMNRx.
An additional 49 SMA patients between the ages of 2 and 12 are needed for the study and we hope to close enrollment soon.
In this study, ISIS - SMNRx was well tolerated at all dose levels in children with SMA.
March 13, 2007 — Cold Spring Harbor, New York — RNA splicing antisense technology studied at Cold Spring Harbor Laboratory (CSHL) effectively corrected an mRNA splicing defect found in spinal muscular atrophy (SMA) patients, and is now ready to be tested in mouse models.
The SMA Foundation funded a pilot study of EIM in children with SMA to determine -LSB-...]
The Phase 1 study of ISIS - SMNRx is a single - 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 - 14 who are medically stable.
ISIS - SMNRx is also being evaluated in an open - label, multiple - dose, dose - escalation Phase 2 study in infants with Type I SMA.
«We see real promise in therapeutic strategies for SMA that increase production of the SMN protein,» said Muscular Dystrophy Association Executive Vice President Research and Medical Director Valerie Cwik, M.D. «We're delighted ISIS Pharmaceuticals is moving forward with a Phase 1 dose - escalation study of its antisense drug in children with SMA.»
This year's ASENT meeting featured a symposium on spinal muscular atrophy (SMA), an inherited motor neuron disease that is the second most common autosomal recessive disorder of children and the most common genetic cause of death in infancy, as a case study in neurology orphan drug development.
CARLSBAD, Calif., February 21, 2013 — Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today announced top - line results from an ongoing open - label, multiple - dose study of ISIS - SMNRx in children with spinal muscular atrophy (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 second study, FIREFISH, is aimed to assess safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy in infants with Type I SMA.
Ongoing radio observations (SMA, JCMT, VLA) of Sirius A are being used to set an observationally determined standard for stellar atmosphere modeling and debris disk studies around A stars, as well as to take the first step toward characterizing potential intrinsic uncertainty in stellar emission at these wavelengths.
NEW YORK, NY May 4, 2012 — The Spinal Muscular Atrophy (SMA) Foundation congratulates the members of the Pilot Study of Biomarkers for Spinal Muscular Atrophy (BforSMA) Trial Group for the recent publication of study results in two articles published by the open - access, peer - reviewed journal, PLoSStudy of Biomarkers for Spinal Muscular Atrophy (BforSMA) Trial Group for the recent publication of study results in two articles published by the open - access, peer - reviewed journal, PLoSstudy results in two articles published by the open - access, peer - reviewed journal, PLoS ONE.
Once we evaluate ISIS - SMNRx as a single - dose in children with SMA, we will move to multiple - doses in our Phase 1 studies and eventually evaluate the drug in Phase 2 studies in children with SMA, including infants with Type I SMA.»
Even while these experiments were proceeding, Dr. Sumner wanted to study the phenomenon of HDAC inhibition in the SMA mouse.
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 studies.
The first hint that HDAC inhibitors might affect SMA came in a 2001 study showing that the weak inhibitor phenylbutyrate increased protein levels in patient cell lines and SMA mice (Proc Natl Acad Sci 2001; 98:9808 — 9813).
Part 2 is a double - blinded, placebo - controlled, randomized confirmatory study in approximately 150 non-ambulant Type II and Type III SMA patients for 24 months.
This study is «a great step forward,» said Chris Lorson, PhD, who studies the molecular basis of SMA.
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 — 671).
A new study implicates a key cellular mechanism as defective in SMA for the first time, providing a new lead for developing future interventions.
Tie is now turning the focus of this study to follow - up observations with JCMT (e.g. POL - 2) as well as many other telescopes (e.g., ALMA, SMA, NRO 45 - m, KVN, FAST 500m...).
The primary objective of this study was to examine the safety and tolerability of SPINRAZA in patients with Type 2 or Type 3 SMA.
Long - term exercise appears to be beneficial for Spinal Muscular Atrophy (SMA) like mice, suggesting a potential of active physiotherapy for patient care; according to a study published today in The Jo
Dr. Pfaff has developed a novel embryonic - stem cell - based model of SMA that phenocopies the pathology of human SMA, and can be used to study the basis of the disease and screen for compounds that might increase survival of motor neurons.
Through these studies he has identified a new transcription factor and associated pathway that could yield new avenues for therapeutic intervention in SMA.