Spinal Muscular Atrophy News and Research

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Spinal muscular atrophy (SMA) is a motor neuron disease and the leading genetic cause of death among infants and toddlers. Characterized by selective loss of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy. Over time, patients afflicted by SMA continue to lose muscle control and strength, leading to progressive inability to walk, stand, sit up and breathe. It is estimated that approximately 1 in 6,000 to 1 in 10,000 infants are born annually worldwide with SMA.

Caliper first quarter revenue increases 25% to $35.8 million

Caliper Life Sciences, Inc., today reported its financial results for the first quarter of 2011. First quarter revenue increased to $35.8 million, or 25%, from $28.7 million in the same period of 2010.

2 May 2011

FightSMA to host panel discussions on spinal muscular atrophy

International non-profit organization FightSMA will be hosting highly respected experts from across the country on Thursday, May 5, 2011 for a series of panel discussions, Thriving with SMA: LIVE!.

30 Apr 2011

Repligen anticipates 30% increase in total revenues for 2011

Repligen Corporation today announced preliminary financial results for fiscal year 2011 which ended March 31, 2011. Total revenue for the year is expected to be $27.3 million compared to $21.0 million in the prior fiscal year, an increase of 30%.

21 Apr 2011

Quest Diagnostics completes Athena Diagnostics acquisition

Quest Diagnostics Incorporated, the world's leading provider of diagnostic testing, information and services, today announced it has completed its previously announced acquisition of Athena Diagnostics, an esoteric laboratory specializing in genetic testing for neurological disorders, from Thermo Fisher Scientific.

From Quest Diagnostics 4 Apr 2011

Enzo Life Sciences introduces ELISA system to detect SMN protein

Enzo Biochem, Inc. and the Spinal Muscular Atrophy Foundation today announced that Enzo's wholly owned subsidiary, Enzo Life Sciences Inc., has launched a unique immunoassay system which can be used for the identification and detection of Survival Motor Neuron protein.

16 Mar 2011

Top-line results from Repligen RG2417 Phase 2b trial results in patients with bipolar depression

Repligen Corporation announced today results of a Phase 2b clinical trial of RG2417, an oral formulation of uridine, in patients with bipolar depression.

8 Mar 2011

Scientists discover new way to generate human motor nerve cells

Scientists have discovered a new way to generate human motor nerve cells in a development that will help research into motor neurone disease.

2 Mar 2011

San Diego Hospice is California's first provider of unique children care program

The Comprehensive Care Management Programs at San Diego Hospice celebrates one year as California's first provider of Partners for Children. This unique state-funded program allows chronically ill children on Medi-Cal and CCS to receive pain and symptom management services, or palliative care, at home.

1 Mar 2011

PsychoGenics establishes alliance with KineMed to provide specialized neurogenesis services

PsychoGenics today announced the establishment of an alliance with KineMed, to provide specialized neurogenesis services to complement PsychoGenics' in vivo testing capabilities.

15 Feb 2011

Repligen third quarter total revenue increases 26% to $7,068,000

Repligen Corporation today reported results for the third quarter of fiscal year 2011, ended December 31, 2010. Total revenue for the quarter was $7,068,000 compared to total revenue of $5,617,000 for the third quarter of fiscal year 2010 ended December 31, 2009, an increase of $1,451,000 or 26%.

9 Feb 2011

Cedars-Sinai receives California Institute grant to develop new technique for specific diseases

A team of scientists from the Cedars-Sinai Regenerative Medicine Institute has been awarded a $1.9 million grant from the California Institute for Regenerative Medicine to fund development of a new technique to aid pharmaceutical discoveries for specific diseases.

3 Feb 2011

RBM, SMA Foundation collaborate to develop plasma protein biomarkers for spinal muscular atrophy

The Spinal Muscular Atrophy Foundation and Rules-Based Medicine, 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 technology platform.

21 Jan 2011

MDA awards Repligen $1.4M for research of new compound with therapeutic potential for SMA

The Muscular Dystrophy Association today announced it has awarded $1.4 million in new research funding to the biopharmaceutical firm Repligen of Waltham, Mass., to complete the preclinical work needed to begin human clinical trials of a promising therapeutic compound for spinal muscular atrophy (SMA).

17 Dec 2010

MDA commits to invest funds in FSMA Quinazoline Program

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.

16 Dec 2010

MDA supports Repligen with $1.4M grant for RG3039 Spinal Muscular Atrophy development program

Repligen Corporation 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.

15 Dec 2010

CSC files IND application to commence Phase I study for Spinal Muscular Atrophy

California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today that CSC has filed an investigational new drug (IND) application with the US Food and Drug Administration (FDA) for approval to commence a Phase I safety study on a jointly-developed stem cell-derived motor neuron transplantation therapy for Spinal Muscular Atrophy (SMA) Type I.

2 Dec 2010

Comprehensive guide to current practice of intraoperative neurophysiology

A new book entitled "Intraoperative Neurophysiologic Monitoring" has been published by Cambridge University Press, co-authored by Nationwide Children's Hospital Pediatric Neurologists Gloria M. Galloway, MD, and Khaled M. Zamel, MD; Marc R. Nuwer, MD, of UCLA; and Jaime R. Lopez, MD of Stanford University.

20 Nov 2010

PROTOX closes first tranche of investment by Warburg Pincus

Protox Therapeutics Inc., a leader in the development of receptor targeted therapeutic fusion proteins, today announced it has closed the first tranche of the investment by Warburg Pincus. The investment was approved by 100% of the votes cast by shareholders at the recent special shareholders meeting.

20 Nov 2010

New computational techniques to overcome obstacles in hard-to-study genes

Scientists have invented methods to scout the human genome's repetitive landscapes, where DNA sequences are highly identical and heavily duplicated. These advances, as reported today in Science, can identify subtle but important differences among people in the number and content of repeated DNA segments.

28 Oct 2010

Critical piece of puzzle in treatment of Spinal Muscular Atrophy

University of Missouri researchers believe they have found a critical piece of the puzzle for the treatment of Spinal Muscular Atrophy (SMA) - the leading genetic cause of infantile death in the world. Nearly one in 6,000 births has SMA, and it is estimated that nearly one in 30 to 40 people have the trait that leads to SMA.

29 Sep 2010