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.
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

New cell type could play an important role in spinal cord injury and disease

New cell type could play an important role in spinal cord injury and disease

Walgreens, MDA team up to offer free seasonal flu shots

Walgreens, MDA team up to offer free seasonal flu shots

WSU researcher receives $418,000 NINDS grant to study spinal muscular atrophy

WSU researcher receives $418,000 NINDS grant to study spinal muscular atrophy

Two ALS-associated genes work in tandem to support long-term survival of motor neurons

Two ALS-associated genes work in tandem to support long-term survival of motor neurons

MDA announces $13.7M in grants to 40 new muscular dystrophy research initiatives

MDA announces $13.7M in grants to 40 new muscular dystrophy research initiatives

NIH/NINDS awards AMRI to develop pre-clinical drug candidate for spinal muscular atrophy

NIH/NINDS awards AMRI to develop pre-clinical drug candidate for spinal muscular atrophy

Repligen first quarter total revenue increases 9% to $7,654,000

Repligen first quarter total revenue increases 9% to $7,654,000

New hope for infants born with Spinal Muscular Atrophy

New hope for infants born with Spinal Muscular Atrophy

FDA grants Fast Track designation for Repligen's RG3039 to treat Spinal Muscular Atrophy

FDA grants Fast Track designation for Repligen's RG3039 to treat Spinal Muscular Atrophy

Repligen total revenue increases 30% to $27,291,000 for fiscal year 2011

Repligen total revenue increases 30% to $27,291,000 for fiscal year 2011

New research on spinal muscular atrophy

New research on spinal muscular atrophy

Evotec, PsychoGenics partner to offer integrated CNS drug discovery solutions

Evotec, PsychoGenics partner to offer integrated CNS drug discovery solutions