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.
Cytokinetics, The ALS Association expand partnership to fight against ALS

Cytokinetics, The ALS Association expand partnership to fight against ALS

Positive data from two studies analyzing Good Start's test for inherited diseases presented at ACOG 2015

Positive data from two studies analyzing Good Start's test for inherited diseases presented at ACOG 2015

Proteomics helps identify previously unrecognized proteins and pathways in nerve regeneration

Proteomics helps identify previously unrecognized proteins and pathways in nerve regeneration

Isis Pharmaceuticals announces positive results from ISIS-ANGPTL3Rx Phase 1 study

Isis Pharmaceuticals announces positive results from ISIS-ANGPTL3Rx Phase 1 study

Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis receives $5 million from Biogen Idec to develop drugs for neurological disorders

Isis receives $5 million from Biogen Idec to develop drugs for neurological disorders

Isis receives $10 million from Biogen Idec to begin IND-supporting studies of ISIS-BIIB4 Rx

Isis receives $10 million from Biogen Idec to begin IND-supporting studies of ISIS-BIIB4 Rx

Isis announces top-line results from ISIS-PTP1B Rx Phase 2 study in type 2 diabetes patients

Isis announces top-line results from ISIS-PTP1B Rx Phase 2 study in type 2 diabetes patients

AMRI awarded NIH contract for drug development services

AMRI awarded NIH contract for drug development services

New computational technique reveals genetic roots of autism, cancers

New computational technique reveals genetic roots of autism, cancers

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Cedars-Sinai to participate in a consortium studying motor neuron disorders

Cedars-Sinai to participate in a consortium studying motor neuron disorders

MU researchers make new breakthrough in spinal muscular atrophy drug

MU researchers make new breakthrough in spinal muscular atrophy drug

Six research institutions awarded NIH grants to create database of human cellular responses

Six research institutions awarded NIH grants to create database of human cellular responses

Splice-switching oligonucleotide drugs alter editing of gene transcript

Splice-switching oligonucleotide drugs alter editing of gene transcript