Sentences with phrase «sma treatment»

Since it has been several months since we last provided a joint update on such activities, we wanted to take a moment to inform you about recent and upcoming government - sponsored, SMA - related research conferences and to provide an update on the SMA Treatment Acceleration Act (H.R. 2149 / S. 1158).

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.

After extensive conversations over the past several weeks with senior staff persons and key Members of Congress who serve on the congressional committees with jurisdiction over the bill, it has become clear that very few, if any, public health bills such as the SMA Treatment Acceleration Act will move through Congress during the lame - duck session set to begin on November 15.

With regard to the SMA Treatment Acceleration Act, despite our best efforts and the unbelievable show of support by families across the nation, the current political climate in Washington makes it highly unlikely that the bill will pass this year.

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.

Not exact matches

SPINRAZA ® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA).

And, more recently, we witnessed the approval of Spinraza as a treatment for spinal muscular atrophy (SMA) in adults and children this past December.

She is passionate about spreading awareness about Selective Mutism and about effective treatment techniques and is excited to contribute to the mission of SMA through her involvement on the Board of Directors.

Through the SMA listserv, professionals share referral information, ask and give guidance to treatment concerns, and share ideas and information.

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

«Our current treatment helps the body create a backup mechanism to combat the disease and extends survival in mice with SMA from just 13 days to a little over five months after only one injection at birth,» Lorson said.

But early approval left medical centers scrambling to establish treatment programs, which became more urgent when the FDA approved the drug for all SMA patients in December 2016, Burgart said.

For now, they wrote, researchers and clinicians will continue to share any new data about nusinersen use in SMA patients to inform future decisions, including how broadly treatment should be pursued.

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.

Early treatment of SMA is crucial for halting muscle degeneration, especially in infants, Burgart said.

His research found that the earlier the treatment can be administered in mice with SMA, the better the outcome.

The SMA model pig was created using a gene therapy approach by knocking down the levels of pig SMN, followed by treatment with human SMN at early and late time points.

Their findings could pave the way for new treatments for spinal muscular atrophy (SMA)-- also known as floppy baby syndrome — which is a leading genetic cause of death in children.

Oligonucleotide treatments recently have been approved by the Food and Drug Administration for two neuromuscular diseases: Duchenne's muscular dystrophy and spinal muscular atrophy (SMA).

«Restoring mitochondrial function might be a new treatment strategy for SMA,» said Yongchao Ma, PhD, senior author and Ann Marie and Francis Klocke, MD Research Scholar, Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago.

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).

New York, NY — November 29, 2004 — The Spinal Muscular Atrophy Foundation, a nonprofit organization dedicated to finding a treatment or cure for spinal muscular atrophy (SMA)-- the leading genetic killer of infants and toddlers — announced that is has funded more than $ 15 million in research in the last 18 months.

«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.

Currently, treatment of SMA is entirely symptomatic and focuses on preserving muscle strength and lung function by physical therapy and assisted ventilation.

Isis is developing ISIS - SMNRx as a potential treatment for all Types of SMA.

SMA seems so ripe for effective treatment that NINDS has developed a model translational research program to accelerate therapy (www.smaproject.org).

Sign the petition for Funding of Spinraza for all Canadian patients affected by Spinal Muscular Atrophy (SMA) Cure SMA Canada, CORD, the Canadian SMA families and our supporters appeal to our provincial health ministers to provide all patients access to the only treatment available for this life threatening disease.

The United States Food and Drug Administration granted Orphan Drug Designation with Fast Track Status to ISIS - SMNRx for the treatment of patients with 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.

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.

Together with the involvement of the SMA Foundation, we now have the opportunity to make a significant impact in the treatment of 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.

The SMA Foundation has diverse drug discovery collaborations with both academia and the private sector to accelerate the development of a treatment or cure.

«Assay Designs is excited and proud to have been selected by the SMA Foundation, and to help enable better understanding and ultimately improved treatment for this debilitating illness,» commented Dan Calvo, President and CEO of Assay Designs.

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

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.

«We are delighted that the SMA Foundation continues to be an active participant in the collaboration as we share a strong commitment to advancing this innovative potential treatment as rapidly as possible.»

The SMA Foundation was established in 2003 by Loren Eng and Dinakar Singh to accelerate the development of a treatment for SMA.

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.

Founded in 2003 to accelerate the development of a treatment for SMA, which afflicts over 25,000 people in the U.S. and has no known treatment, the SMA Foundation works with leaders in research, industry and government to raise awareness, support and research investment.

Walenbergh SMA, Houben T, Hendrikx T, Jeurissen MLJ, Gorp van PJ, Vaes N, Olde Damink SWM, Verheyen F, Koek GH, Lütjohann D, Grebe A, Latz E, Shiri - Sverdlov R. Weekly Treatment of 2 - Hydroxypropyl - β - cyclodextrin Improves Intracellular Cholesterol Levels in LDL Receptor Knockout Mice.

SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

In a group of later - onset SMA patients (mean treatment exposure 34 months), 69 % had elevated urine protein.

Loren Eng, Co-Founder and President of the SMA Foundation commented, «This network is a critical and necessary step in developing treatments for spinal muscular atrophy.

About SMA Foundation The SMA Foundation is a nonprofit organization, founded in 2003, dedicated to finding a treatment and potential cure for Spinal Muscular Atrophy (SMA).

It is the hope and desire of each of the contributing organizations that this effort will accelerate the development of a treatment for SMA, a devastating neurodegenerative disease that leads to death by age two in up to 50 % of patients.

Obtaining approval for your patient's treatment for spinal muscular atrophy (SMA) often requires your practice or facility to complete several steps, including conducting a thorough Benefit Investigation to determine if an authorization and / or medical exception is needed prior to treatment approval.

While some symptoms of SMA can be alleviated, there is currently no treatment that can change the course of the disease.

The research network augments the Foundation's aggressive agenda to fund research initiatives which will accelerate the development of a treatment or cure for SMA.

«We are delighted to support the efforts of the SMA Foundation to develop better treatments for this terrible disease,» said Craig Benson, Chief Executive Officer of RBM.