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.
New understanding of how motor neurons die in spinal muscular atrophy patients

New understanding of how motor neurons die in spinal muscular atrophy patients

Innovative strategy for regenerating skeletal muscle tissue

Innovative strategy for regenerating skeletal muscle tissue

DYNC1H1 genetic mutation contributes to SMA-LED

DYNC1H1 genetic mutation contributes to SMA-LED

New system measures strength of communication between muscle cells and motor neurons

New system measures strength of communication between muscle cells and motor neurons

Biogen Idec first quarter revenues increase 7% to $1.3 billion

Biogen Idec first quarter revenues increase 7% to $1.3 billion

Couples with infertility and miscarriages offered detailed embryo screening with 99% accuracy

Couples with infertility and miscarriages offered detailed embryo screening with 99% accuracy

Positive results from Repligen's RG3039 Phase 1 study for spinal muscular atrophy

Positive results from Repligen's RG3039 Phase 1 study for spinal muscular atrophy

Abnormally low level of SMN protein linked to movement problems in spinal muscular atrophy

Abnormally low level of SMN protein linked to movement problems in spinal muscular atrophy

Repligen total revenue increases to $7,165,000 for quarter ended December 31, 2011

Repligen total revenue increases to $7,165,000 for quarter ended December 31, 2011

Drug companies forge joint ventures for smart pills

Drug companies forge joint ventures for smart pills

Biogen Idec 2011 total revenues increase 7% to $5.0 billion

Biogen Idec 2011 total revenues increase 7% to $5.0 billion

Pediatric research project to expand data collection linked to newborn screening results

Pediatric research project to expand data collection linked to newborn screening results

BioTime to market muscle progenitor cell lines bearing hereditary diseases

BioTime to market muscle progenitor cell lines bearing hereditary diseases

Einstein, Montefiore receive NINDS grant to establish clinical site for NeuroNEXT

Einstein, Montefiore receive NINDS grant to establish clinical site for NeuroNEXT

PTC, Roche announce licensing agreement for SMA programme

PTC, Roche announce licensing agreement for SMA programme

Targeting synthetic RNA to specific gene could help reduce severity of SMA

Targeting synthetic RNA to specific gene could help reduce severity of SMA

SNI receives NINDS grant to conduct clinical research studies on brain disorders

SNI receives NINDS grant to conduct clinical research studies on brain disorders

Cancer cells maintain rapid proliferation due to PK-M2 enzyme

Cancer cells maintain rapid proliferation due to PK-M2 enzyme

Communication breakdown between nerves and muscles in SMA mouse model

Communication breakdown between nerves and muscles in SMA mouse model

Cedars-Sinai inaugurates new facility to generate induced pluripotent stem cells

Cedars-Sinai inaugurates new facility to generate induced pluripotent stem cells