Enzo Biochem Unit Launches First-to-Market Survival Motor Neuron (SMN) Protein Immunoassay System

March 16, 2011

Enzo Biochem Unit Launches First-to-Market Survival Motor Neuron (SMN) Protein Immunoassay System

Press Release | March 16, 2011

Enzo Biochem, Inc. and the Spinal Muscular Atrophy (SMA) Foundation today announced that Enzo’s wholly owned subsidiary, Enzo Life Sciences Inc., has launched a unique immunoassay (ELISA) system which can be used for the identification and detection of Survival Motor Neuron (SMN) protein. The kit is the result of a collaborative agreement between Enzo and the SMA Foundation.for the development of reagents and assays for SMN protein. The availability of an effective SMN ELISA could further enable and expedite drug discovery, development and therapy for Spinal Muscular Atrophy, the leading genetic cause of mortality in infants and toddlers.

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Quest Diagnostics to Acquire Athena Diagnostics, Establishing Leading Position in Rapidly Growing Neurology Testing Market

February 25, 2011

Quest Diagnostics to Acquire Athena Diagnostics, Establishing Leading Position in Rapidly Growing Neurology Testing Market

Press Release | February 25, 2011

MADISON, N.J., Feb. 24, 2011 /PRNewswire via COMTEX/ — Quest Diagnostics Incorporated (NYSE: DGX), the world’s leading provider of diagnostic testing, information and services, announced today that it has signed a definitive agreement to acquire Athena Diagnostics, an esoteric laboratory specializing in genetic testing for neurological disorders, from Thermo Fisher Scientific (NYSE: TMO) for $740 million in cash.

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Study with Funding from FSMA Shows a Muscle Specific Intervention Improves Phenotype in a Mouse Model of SMA

February 18, 2011

Study with Funding from FSMA Shows a Muscle Specific Intervention Improves Phenotype in a Mouse Model of SMA

Press Release | February 18, 2011

Dr. Charlotte Sumner and colleagues at Johns Hopkins University publish a study in the journal Human Molecular Genetics showing improvement in survival but not motor function in a severe model of Spinal Muscular Atrophy by increasing the levels of the muscle modulator Insulin-like growth factor 1 (IGF-1).

Spinal muscular atrophy (SMA) is an inherited motor neuron disease caused by the mutation of the survival motor neuron 1 (SMN1) gene and deficiency of the SMN protein. Severe SMA mice have abnormal motor function and small, immature myofibers early in development suggesting that SMN protein deficiency results in retarded muscle growth. Insulin-like growth factor 1 (IGF-1) stimulates myoblast proliferation, induces myogenic differentiation, and generates myocyte hypertrophy in vitro and in vivo.

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FightSMA Marks 20th Birthday with New Home on the Web

February 18, 2011

FightSMA Marks 20th Birthday with New Home on the Web

Press Release | February 18, 2011

(Richmond, Virginia) – Parents fighting spinal muscular atrophy (SMA), the leading genetic killer of children under two, need help and information. Recognizing that need, FightSMA, an international nonprofit organization dedicated to finding a cure for SMA, is announcing the launch of its all new website, FightSMA.org.

“When we set out to redesign FightSMA.org, we looked at the enormous amount of information we have for SMA parents, and hoped to make it more accessible and easy to find,” said FightSMA co-founder and president, Martha Slay. “The new website features better categorization and a more flexible design that will help us in our fight, and a new website is a great way to celebrate our birthday!”

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New Mouse Models of SMA Developed Using Mouse Smn Genes that Mimic Human SMN2

February 2, 2011

New Mouse Models of SMA Developed Using Mouse Smn Genes that Mimic Human SMN2

FSMA (Press Release) | January 31, 2011

Dr. Christine DiDonato and colleagues at Children’s Memorial Research Center in Chicago, IL  and Ottawa Health Research Institute in Ottawa, ON recently published 2 new mouse models of SMA in the Journal PLoS One with funding from Families of SMA.

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SMA Foundation and Rules-Based Medicine Collaborate on Biomarker Panels to Guide SMA Therapeutic Development

January 19, 2011

SMA Foundation and Rules-Based Medicine Collaborate on Biomarker Panels to Guide SMA Therapeutic Development

Press Release | January 19, 2011

NEW YORK and AUSTIN, Texas, Jan. 19, 2011 /PRNewswire/ — The Spinal Muscular Atrophy (SMA) Foundation and Rules-Based Medicine, Inc. (RBM) announced today that they have reached the first milestone in a program to develop a panel of plasma protein biomarkers for SMA using RBM’s Multi-Analyte Profiling (MAP) technology platform.  In this collaboration, RBM will discover and also confirm plasma protein biomarker candidates previously identified from the multicenter Biomarkers for SMA (BforSMA) clinical study sponsored by the SMA Foundation and conducted by BG Medicine. The identification of such biomarkers may help to assess drug efficacy and shorten the duration of clinical trials of SMA therapies.

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Families of Spinal Muscular Atrophy Evaluating Six New Drug Discovery Programs for New Funding in 2011

December 29, 2010

Families of Spinal Muscular Atrophy Evaluating Six New Drug Discovery Programs for New Funding in 2011

Press Release | December 29, 2010

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.   Our Translational Advisory Committee (TAC) reviewed and then selected six of these for further consideration for funding.   Each of these six applicants will now submit a full detailed proposal, which will be due in February 2011.

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FSMA is Proud to be Implementing a New Support Program for Newly Diagnosed SMA Families

December 17, 2010

FSMA is Proud to be Implementing a New Support Program for Newly Diagnosed SMA Families

Press Release | December 16, 2010

Everyone at Families of SMA is extremely proud to be implementing our latest program for newly diagnosed SMA families. We have had several conversations regarding better ways to support families and those that are newly diagnosed.  Thanks to Hillary & Keith Schmid, the FSMA Greater Florida Chapter and Audra & Andy Butler, we are excited to announce that Families of SMA has created a binder of information with specific resources for each state. These binders contain important information about SMA, as well as provide families with a list of available resources within their state.  The binders can also act as an organized place for families to keep important medical information concerning their child.

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FSMA Research Funding Leads to the Publication of 17 Journal Articles in 2010

December 17, 2010

FSMA Research Funding Leads to the Publication of 17 Journal Articles in 2010

Press Release | December 16, 2010

The research goal at Families of Spinal Muscular Atrophy is to accelerate the discovery and development of a treatment and cure for SMA. To help achieve this we fund basic research grants, along with later-stage drug discovery programs and clinical trials. FSMA research funding contributed to the results in 17 published articles in 2010.

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Repligen Receives $1.4 Million Research Grant from the MDA to Support SMA Development Program

December 15, 2010

Repligen Receives $1.4 Million Research Grant from the Muscular Dystrophy Association to Support Spinal Muscular Atrophy Development Program

Repligen Corporation Press Release | December 15, 2010

WALTHAM, Mass., Dec. 15, 2010 /PRNewswire/ — Repligen Corporation (Nasdaq: RGEN) today announced that the Company has received $1,400,000 in research funding from the Muscular Dystrophy Association (“MDA”) to support the ongoing development of RG3039 for Spinal Muscular Atrophy (“SMA”).  RG3039, our lead compound, is an inhibitor of an RNA processing enzyme which targets increased production of SMN, a protein of deficient levels in patients with SMA.  The goal of this grant is to support the advancement of RG3039 into human clinical testing, including continued evaluation in preclinical models of SMA, GMP manufacturing for human clinical trials and early clinical evaluation in healthy volunteers and patients.  Funding from the Muscular Dystrophy Association provides important support for Repligen’s development programs and access to a global network of scientists, physicians and patients.  Repligen has previously been awarded two research grants from the MDA to support its Friedreich’s ataxia program.

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