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SMA Foundation and PTC Therapeutics Expand Partnership and Commit to an Initial Investment of Up to $60M to Advance Drug Discovery and Development Research in Regenerative Medicine

March 9, 2021

The Spinal Muscular Atrophy (SMA) Foundation and PTC Therapeutics have entered a new collaboration focused on regenerative medicine to further advance scientific research in SMA and other neuromuscular disorders. Recent FDA approvals of several disease modifying therapies for SMA has made regenerative medicine approaches the next frontier in drug discovery and development. The SMA Foundation-PTC…

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The Spinal Muscular Atrophy Foundation Rings Nasdaq Opening Bell

August 18, 2018

The Spinal Muscular Atrophy (SMA) Foundation, a non-profit, organization whose mission is to accelerate the development of treatments for SMA, the leading cause of genetic death in young children, visited the Nasdaq MarketSite in Times Square on August 17, 2018. In honor of the occasion, Dinakar Singh, Co-Founder and Chairman of the Board of Directors,…

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Congratulations to Dr. Brian McCabe and Dr. Livio Pellizzoni for Their Recent Publications in Cell

October 11, 2012

Congratulations to Dr. Brian McCabe and Dr. Livio Pellizzoni of Columbia University’s Motor Neuron Center for their publications in Cell. The first study, led by Dr. McCabe, shows that SMN restoration in proprioceptive neurons and interneurons corrects defects in motor neurons and muscles in a fly SMA model. Surprisingly, SMN restoration in muscles or motor…

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President Obama Signs into Law The Food and Drug Administration Safety and Innovation Act (FDASIA)

July 10, 2012

On July 9, 2012, President Obama signed into law The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187. In their news release, the EveryLife Foundation for Rare Diseases highlights a key aspect of this legislation as pertinent to SMA drug development —  it will “empower [the] FDA to use all the science…

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The Spinal Muscular Atrophy Foundation Announces the Publication of Results from the Biomarkers for SMA (BforSMA) Clinical Study

May 4, 2012

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 goal of the BforSMA study was…

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The Spinal Muscular Atrophy Foundation Announces a Biomarker Panel to Guide SMA Therapeutic Development

April 3, 2012

NEW YORK, NY | April 3, 2012 – The Spinal Muscular Atrophy (SMA) Foundation announced today the launch of a biomarker assay panel for SMA using Myriad RBM’s Multi-Analyte Profiling (MAP) technology platform. The SMA-MAP panel is designed to evaluate the severity of SMA and disease progression and can be used to assess drug efficacy…

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NORD Supports FAST Act Introduced by Representatives Stearns and Towns

March 9, 2012

Two bills have been introduced in Congress which are intended to expedite the drug approval process for diseases with serious unmet medical need such as SMA. On February 15, 2012, the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act, S. 2113, was introduced by Senator Kay Hagan (D-NC) and on March 5, 2012…

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SMA Foundation Observes Rare Disease Day

February 21, 2012

New York, NY, February 21, 2012 – The Spinal Muscular Atrophy (SMA) Foundation will join the National Organization for Rare Disorders (NORD) and others around the world in observing Rare Disease Day on February 29.  The purpose is to focus attention on the needs of patients and families affected by rare diseases. “This is a…

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Roche, PTC & SMA Foundation Agreement to Develop a Treatment for SMA Receives Extensive Coverage

February 13, 2012

On November 29, 2011, Roche, PTC Therapeutics and the SMA Foundation announced the signing of an agreement to develop a treatment for Spinal Muscular Atrophy. Several media sources reported on the agreement including BioCentury, BioWorld Today, Bloomberg Businessweek, Chemical & Engineering News, Clinical Professionals, Dow Jones VentureWire, Drug Discovery News, FierceBiotech, Genetic Engineering and Biology News,…

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Isis Initiates Phase 1 Clinical Study of ISIS-SMNRx in Patients with Spinal Muscular Atrophy

December 19, 2011

CARLSBAD, Calif., December 19, 2011 – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has initiated a Phase 1 study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA). SMA is a severe motor-neuron disease that is the leading genetic cause of infant mortality. Isis is developing ISIS-SMNRx as a potential treatment for all…

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Roche Signs Agreement with PTC Therapeutics to Advance Treatment for Spinal Muscular Atrophy (SMA)

November 29, 2011

Collaboration offers new hope for a potential treatment for the leading genetic cause of death in infants and toddlers Basel, Switzerland, South Plainfield, NJ, USA and New York, NY, USA – November 29, 2011 – Roche (SIX: RO, ROG; OTCQX: RHHBY), PTC Therapeutics, Inc. (PTC) and the SMA Foundation, announced today a licensing agreement for…

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Congratulations to Stephen Mikita, Esq., on his new role as a Patient Representative to the FDA

November 23, 2011

Congratulations to SMA Foundation Board Member, Stephen Mikita, Esq., on his new role as a Patient Representative to the U.S. Food and Drug Administration (FDA). Mr. Mikita recently completed the Patient Representative training program with the FDA’s Office of Special Health Issues. As a Patient Representative, Mr. Mikita will participate in advisory committees to review…

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