Sentences with phrase «sma trials»

Other HDAC inhibitors are in development for cancer treatment, and these, rather than trichostatin A, are more likely candidates for human SMA trials in the future.

For more information on other SMA trials and the International SMA Patient Registry is recruiting for, please see the press release below.

Beyond providing important information for SMA trial planning going forward, their results also highlight the importance of carefully validating specific biomarkers in a preclinical trial situation.

Kids in the Ionis SMA trial did so well that regulators asked them to halt the trial early, so that every kid in the study, including those getting the placebo, could receive the drug.

Not exact matches

The results were significant enough to prompt the FDA to halt the trial early, in August 2016, and expand access to all SMA - 1 patients.

Several SMA treatment trials are in progress, but the community is especially tracking a single - dose gene therapy that, if approved, would have its own cost and treatment implications.

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.

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-...]

«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 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 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.

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.

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, PLoS ONE.

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

Nonetheless, SMA parents are solidly behind future trials.

Dr. Swoboda led a trial of valproic acid in human SMA, whose results are due out soon.

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.

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 SMA Foundation is taking a bold initiative to identify markers of disease severity in spinal muscular atrophy,» noted Richard Finkel, MD, a principal investigator for the trial at the Children's Hospital of Philadelphia.

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

«We are now able to make biochemical measurements that will help us objectively assess functional changes in children and adults with SMA, and, hopefully, detect early signals of therapeutic success in new drug trials for this devastating disease.»

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 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.

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

«This Phase 1 trial in children with SMA Type 2 will allow us to evaluate safety, optimal dosing and proof - of - concept for efficacy of AVXS - 101 compared to the well - characterized natural history using the one - time intrathecal route of administration,» said Dr. Sukumar Nagendran, Chief Medical Officer of AveXis.

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 administraTrial 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 administratrial — 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.

«We are quite pleased to initiate our first trial of AVXS - 101 in patients with SMA Type 2,» said Sean Nolan, President and Chief Executive Officer of AveXis.

The trial will enroll 27 infants and children with a genetic diagnosis consistent with SMA, including the bi-allelic deletion of SMN1 and three copies of SMN2 without the SMN2 genetic modifier, who are able to sit but have no historical or current ability to stand or walk.

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.

There was no proof it would help (trials are under way at Stanford, partly sponsored by the SMA Foundation), but it had been safely used before on kids with sickle - cell anemia.

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.

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.

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

SMA VALIANT Trial: A prospective, double - blind, placebo controlled trial of valproic acid in ambulatory adults with spinal muscular atrTrial: A prospective, double - blind, placebo controlled trial of valproic acid in ambulatory adults with spinal muscular atrtrial of valproic acid in ambulatory adults with spinal muscular atrophy.

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).

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.