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By Cure SMA
Cure SMA has announced a new partnership with the District Policy Group, a boutique lobbying, public policy, and advocacy practice, to advocate for several key policy areas on behalf of the SMA community.
By Cure SMA
The SMA drug pipeline has grown dramatically in just over a decade. Of the 17 programs in the pipeline, seven of these are in clinical trials, and two of those are in Phase 3 clinical trials.
By Cure SMA
Cure SMA, the leading organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), recently provided an additional $445,000 grant toward a gene therapy for SMA at Nationwide Children’s Hospital.
By Cure SMA
Cure SMA has awarded a $140,000 research grant to Chad Heatwole, MD, at the University of Rochester for his project, "Development of a Clinically Relevant Outcome Measure for SMA Therapeutic Trials." This grant is supported by funding from The Spinal...
By Cure SMA
Cure SMA recently announced a new $315,000 drug discovery grant to the California Institute of Biomedical Research (CALIBR).
By Cure SMA
Cure SMA has awarded an $80,000 research grant to Linda Lowes, PT, PhD, at Nationwide Children’s Hospital for her project, “Development of An Innovative Outcome Measure to Define Disease Progression in SMA Type I for Use in the Home or Clinic.” As...
By Cure SMA
Spinal muscular atrophy (SMA) is the number one genetic cause of death for infants, and 1 in 50 people carry the genetic mutation that causes SMA. But even so, many people are not familiar with SMA. Thanks to the Cure SMA community, that is changing.
By Cure SMA
Cure SMA has awarded a $140,000 research grant to Mustafa Sahin, MD, PhD, at Boston Children’s Hospital for his project, “mTOR and Protein Synthesis in SMA.” Individuals with spinal muscular atrophy don’t produce survival motor neuron (SMN)...
By Cure SMA
Cure SMA has awarded a $140,000 research grant to Francesco Lotti, PhD, at Columbia University, for his project, "Role of Sumoylation in SMN Function and SMA Pathology." Survival motor neuron (SMN) protein is critical to the function of the nerves that...
By Cure SMA
Cure SMA, the leading organization in the US dedicated to the treatment and cure of spinal muscular atrophy (SMA), has issued a statement commending the US House of Representatives Energy and Commerce Committee for their release of the 21st Century Cures.
By Cure SMA
Cure SMA has announced a new $50,000 clinical care grant to Dr. Nilesh Mehta, for his project focusing on nutrition in spinal muscular atrophy (SMA).
By Cure SMA
Audrey Lewis founded Families of SMA, now Cure SMA, 30 years ago.
By Karen Wheeler
If you think you have limitations - STOP -There are no limitations when it comes to imagination.
By Families of SMA
This is the fourth drug discovery project funded by Families of SMA in 2013 with a total investment of $550,000.
By Families of SMA
This funding will build on the consensus statement for the standard of care for SMA with data driven results on specific areas of SMA care such as respiratory, nutritional, and surgical interventions.
By Families of SMA
This three-year multi-million dollar cooperative agreement to Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital in the amount of $3,752,462, funds pre-clinical drug...
By Families of SMA
Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen’s spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA).
By Families of SMA
The SMA community now has 3 clinical trials testing new SMA therapies, and an additional 10 programs in earlier stages of the drug development pipeline.
By FSMA
Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA). Isis is developing ISIS-SMNRx as a potential treatment for all types of SMA.
By Families of SMA
Awards to help develop new therapies for SMA. These new awards encompass $745,000 for the basic research portion of the $3 Million in new research funding that FSMA announced earlier this year.

All Press Releases

By Cure SMA
Cure SMA has announced a new partnership with the District Policy Group, a boutique lobbying, public policy, and advocacy practice, to advocate for several key policy areas on behalf of the SMA community.
By Cure SMA
The SMA drug pipeline has grown dramatically in just over a decade. Of the 17 programs in the pipeline, seven of these are in clinical trials, and two of those are in Phase 3 clinical trials.
By Cure SMA
Cure SMA, the leading organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), recently provided an additional $445,000 grant toward a gene therapy for SMA at Nationwide Children’s Hospital.
By Cure SMA
Cure SMA has awarded a $140,000 research grant to Chad Heatwole, MD, at the University of Rochester for his project, "Development of a Clinically Relevant Outcome Measure for SMA Therapeutic Trials." This grant is supported by funding from The Spinal...
By Cure SMA
Cure SMA recently announced a new $315,000 drug discovery grant to the California Institute of Biomedical Research (CALIBR).
By Cure SMA
Cure SMA has awarded an $80,000 research grant to Linda Lowes, PT, PhD, at Nationwide Children’s Hospital for her project, “Development of An Innovative Outcome Measure to Define Disease Progression in SMA Type I for Use in the Home or Clinic.” As...
By Cure SMA
Spinal muscular atrophy (SMA) is the number one genetic cause of death for infants, and 1 in 50 people carry the genetic mutation that causes SMA. But even so, many people are not familiar with SMA. Thanks to the Cure SMA community, that is changing.
By Cure SMA
Cure SMA has awarded a $140,000 research grant to Mustafa Sahin, MD, PhD, at Boston Children’s Hospital for his project, “mTOR and Protein Synthesis in SMA.” Individuals with spinal muscular atrophy don’t produce survival motor neuron (SMN)...
By Cure SMA
Cure SMA has awarded a $140,000 research grant to Francesco Lotti, PhD, at Columbia University, for his project, "Role of Sumoylation in SMN Function and SMA Pathology." Survival motor neuron (SMN) protein is critical to the function of the nerves that...
By Cure SMA
Cure SMA, the leading organization in the US dedicated to the treatment and cure of spinal muscular atrophy (SMA), has issued a statement commending the US House of Representatives Energy and Commerce Committee for their release of the 21st Century Cures.
By Cure SMA
Cure SMA has announced a new $50,000 clinical care grant to Dr. Nilesh Mehta, for his project focusing on nutrition in spinal muscular atrophy (SMA).
By Cure SMA
Audrey Lewis founded Families of SMA, now Cure SMA, 30 years ago.
By Karen Wheeler
If you think you have limitations - STOP -There are no limitations when it comes to imagination.
By Mary Ann Liebert, Inc., publishers
To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA.
By Families of SMA
This is the fourth drug discovery project funded by Families of SMA in 2013 with a total investment of $550,000.
By Families of SMA
This funding is being awarded to Co-Principal Investigators Dr. Arthur Burghes at Ohio State University and Dr. Christian Lorson at University of Missouri.
By Families of SMA
15 new drugs now in development for SMA. 3 in active clinical trials. 11 companies investing in SMA drug programs.
By Families of SMA
This funding will build on the consensus statement for the standard of care for SMA with data driven results on specific areas of SMA care such as respiratory, nutritional, and surgical interventions.
By Families of SMA
This three-year multi-million dollar cooperative agreement to Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital in the amount of $3,752,462, funds pre-clinical drug...
By Families of SMA
Dr. Rubin's project at Harvard is the first of three drug discovery awards that will be given by Families of SMA in the next few months.
By Families of SMA
Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen’s spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA).
By Families of SMA
Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program.
By Families of SMA
Continued investment in basic research leads to greater understanding of the exact nature, causes, and consequences of SMA.
By Families of SMA
This new research funding will support a project at Northwestern University to better understand when and where SMN protein is needed in less severe SMA.
By Families of SMA
This new research funding will support a project at Indiana University School of Medicine to better understand the role of SMN protein in motor axons.
By Families of SMA
This new research funding will support a project at the University of Southern California to better understand the role of astrocytes in SMA disease pathology.
By FSMA
This new research funding will support a project at Johns Hopkins University to learn more about motor axon growth in mouse models of SMA.
By Families of SMA
The SMA community now has 3 clinical trials testing new SMA therapies, and an additional 10 programs in earlier stages of the drug development pipeline.
By Families of SMA
Repligen Corporation has initiated a Phase 1b clinical study of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy (SMA).
By Families of SMA
Families of Spinal Muscular Atrophy (FSMA) is dedicated to creating a treatment and cure for Spinal Muscular Atrophy (SMA) by funding and advancing a comprehensive research program.
By Families of SMA
Families of SMA is pleased to announce the award of up to $750,000 for an important new grant to Dr. Brian Kaspar at Nationwide Children’s Hospital.
By Families of SMA
The 12th annual Concert for a Cure event to be held on May 12th, 2012 is aiming to reach the incredible milestone of raising $1,000,000 for Spinal Muscular Atrophy (SMA) Research.
By Families of SMA
Repligen Corporation (NASDAQ:RGEN) today announced positive results from a Phase 1 study to evaluate the pharmacokinetic (PK) and safety profile of RG3039, a novel small molecule drug candidate for the potential treatment of SMA.
By Families of SMA
With the 9th Annual Jacob’s Run, Walk & Roll Event to be held on April 1st, The Jacob Isaac Rappoport Foundation is aiming to reach the milestone of raising $1,000,000 for Spinal Muscular Atrophy (SMA) Research.
By Families of SMA
Developing new therapies to treat and ultimately cure SMA is the driving force behind Families of SMA. To achieve this goal, Families of SMA aggressively invests in drug development research.
By Families of SMA
Nutrition Basics: Fostering Health and Growth for Spinal Muscular Atrophy is Families of SMA’s newest release of the SMA Care Series Booklets, which are provided free of charge to all SMA families, as well as SMA Care Providers worldwide.
By FSMA
Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA). Isis is developing ISIS-SMNRx as a potential treatment for all types of SMA.
By Stylish Stagers, Inc.
Dream Room Makeovers, Inc with Stylish Stagers, Inc. to Remodel Union, NJ Home in One Day!
By Families of SMA
Awards to help develop new therapies for SMA. These new awards encompass $745,000 for the basic research portion of the $3 Million in new research funding that FSMA announced earlier this year.
By Mama Bracelets, LLC
Mama Bracelets' owners launch SMA Awareness bracelets in efforts to fund research to cure SMA as their daughter has been recently diagnosed with SMA Type 2.
By FSMA
The new funding awards will be allocated into three distinct research areas: 1)Basic Research to understand the disease, 2)Drug Discovery to develop new SMA therapies, and 3)Clinical Research to provide the means to test new drugs effectively.
By Families of SMA
Boynton Beach residents Shaina and Adi Rappoport have served as the event organizers since 2002 when their son, Jacob, passed way from SMA (Spinal Muscular Atrophy) related complications. Jacob was nine months old.
By Families of SMA
As 2010 comes to a close, we wanted to thank you for your donations and efforts that have supported our research programs and family services.
By Families of SMA
In response to our October Request for Proposals (RFP) for new drug programs to develop therapies for Spinal Muscular Atrophy we received nine Letters of Intent for potential programs.
By Families of SMA
The MDA has committed to invest funds in the FSMA Quinazoline Program at Repligen Corporation to assist in advancing the program to an IND filing with the FDA. FSMA began the Quinazoline, or DcpS inhibitor RG3039, program in 2000.
By Kids Wish Network
A boy suffering from spinal muscular atrophy has his wish to visit the Florida theme parks granted through national children’s charity Kids Wish Network.
By Families of SMA
Families of SMA announces Request for Proposals (RFP) for new drug programs to develop therapies for Spinal Muscular Atrophy. FSMA is aiming to fund two new preclinical drug development programs in 2011, with more to follow.
By Families of Spinal Muscular Atrophy
All across the United States volunteers will come together to raise funds for critical SMA research and patient support programs.
By Fight SMA
Richmond-based FightSMA announced today a new fundraising campaign for spinal muscular atrophy gene therapy, “Realizing the Dream.”
By Fight SMA
A nonprofit dedicated to developing a treatment for the deadly childhood crippler spinal muscular atrophy (SMA), is awarding a $250,000 research grant to Dr. Brian Kaspa of The Ohio State University.


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