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.
Simple, five minute saliva test to determine baby's risk for more than 100 life-threatening genetic diseases

Simple, five minute saliva test to determine baby's risk for more than 100 life-threatening genetic diseases

PTC Therapeutics initiates additional clinical trial of ataluren in boys and young men with nmDBMD

PTC Therapeutics initiates additional clinical trial of ataluren in boys and young men with nmDBMD

A novel approach for funding children's genetic disease research

A novel approach for funding children's genetic disease research

CSC completes a Series B financing round

CSC completes a Series B financing round

CSC's stem cell-derived motor neuron product for SMA receives FDA orphan drug designation

CSC's stem cell-derived motor neuron product for SMA receives FDA orphan drug designation

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

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

Second-quarter fiscal 2010 results announced by Repligen

Second-quarter fiscal 2010 results announced by Repligen

Tetracycline-like compound raises hope for treatment of spinal muscular atrophy

Tetracycline-like compound raises hope for treatment of spinal muscular atrophy

Families of Spinal Muscular Atrophy and Repligen partner to develop a treatment for SMA

Families of Spinal Muscular Atrophy and Repligen partner to develop a treatment for SMA

Intellectual property covering compounds that have utility in treating SMA licensed

Intellectual property covering compounds that have utility in treating SMA licensed

iPierian to participate in $3.7 million NIH grant awarded to The Johns Hopkins University

iPierian to participate in $3.7 million NIH grant awarded to The Johns Hopkins University

Multi-million dollar cooperative agreement for SMA drug development program

Multi-million dollar cooperative agreement for SMA drug development program

Safeway raises funds to support neuromuscular disease medical research and therapies

Safeway raises funds to support neuromuscular disease medical research and therapies

Psychogenics and Astrazeneca sign agreement to identify new compounds for treating Central Nervous System disorders

Psychogenics and Astrazeneca sign agreement to identify new compounds for treating Central Nervous System disorders

Office of Orphan Products Development of the FDA grants Orphan Drug Designation to Quinazoline495

Office of Orphan Products Development of the FDA grants Orphan Drug Designation to Quinazoline495

Discovery of potential treatment for spinal muscular atrophy

Discovery of potential treatment for spinal muscular atrophy

Rare window on spinal muscular atrophy genetics

Rare window on spinal muscular atrophy genetics

Scientists make electrically active motor neurons from iPS cells

Scientists make electrically active motor neurons from iPS cells

Discovery of target that could ease spinal muscular atrophy symptoms

Discovery of target that could ease spinal muscular atrophy symptoms

Patient-derived induced stem cells retain disease traits

Patient-derived induced stem cells retain disease traits