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Study findings highlight importance of early detection of SMA through newborn screening

Study findings highlight importance of early detection of SMA through newborn screening

Investigational drug may help increase protein levels in babies with spinal muscular atrophy

Investigational drug may help increase protein levels in babies with spinal muscular atrophy

Johns Hopkins researchers identify new biological target for treating spinal muscular atrophy

Johns Hopkins researchers identify new biological target for treating spinal muscular atrophy

Promising new therapeutic target could lead to better prognosis of spinal muscular atrophy

Promising new therapeutic target could lead to better prognosis of spinal muscular atrophy

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

MU researchers make new breakthrough in spinal muscular atrophy drug

MU researchers make new breakthrough in spinal muscular atrophy drug

Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

Joint research program in SMA enters initial stage of clinical development

Joint research program in SMA enters initial stage of clinical development

Research Institute receives Fast Track status for its gene therapy product for treatment of SMA

Research Institute receives Fast Track status for its gene therapy product for treatment of SMA

Researchers develop TSUNAMI method to create animal model of adult-onset version of SMA

Researchers develop TSUNAMI method to create animal model of adult-onset version of SMA

Two CUMC studies provide new insights into spinal muscular atrophy

Two CUMC studies provide new insights into spinal muscular atrophy

Investigators discover biological explanation for why low levels of oxygen advance SMA symptoms

Investigators discover biological explanation for why low levels of oxygen advance SMA symptoms

PTC, Roche announce licensing agreement for SMA programme

PTC, Roche announce licensing agreement for SMA programme

Repligen receives approval from FDA to commence RG3039 Phase 1 trial in Spinal Muscular Atrophy

Repligen receives approval from FDA to commence RG3039 Phase 1 trial in Spinal Muscular Atrophy

Enzo Life Sciences introduces ELISA system to detect SMN protein

Enzo Life Sciences introduces ELISA system to detect SMN protein

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

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

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

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

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

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

CSHL scientists reverse symptoms of Type III SMA using ASOs

CSHL scientists reverse symptoms of Type III SMA using ASOs

ISIS-FXIRx and ISIS-SMNRx included in Isis Pharmaceuticals' development pipeline

ISIS-FXIRx and ISIS-SMNRx included in Isis Pharmaceuticals' development pipeline