Spinal Muscular Atrophy News and Research

RSS
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.
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

New paediatric exoskeleton aims to support children with spinal muscular atrophy

New paediatric exoskeleton aims to support children with spinal muscular atrophy

Bereaved parents feel that pediatric end-of-life care needs improvement

Bereaved parents feel that pediatric end-of-life care needs improvement

Gene-replacement therapy could be potential treatment option for SMARD1

Gene-replacement therapy could be potential treatment option for SMARD1

Long-term exercise may benefit Spinal Muscular Atrophy patients

Long-term exercise may benefit Spinal Muscular Atrophy patients

Dr. Elliot J. Androphy receives IGNITE grant to develop drug candidate for spinal muscular atrophy

Dr. Elliot J. Androphy receives IGNITE grant to develop drug candidate for spinal muscular atrophy

Neuroscientists identify specific enzyme that plays critical role in spinal muscular atrophy

Neuroscientists identify specific enzyme that plays critical role in spinal muscular atrophy

Synthetic RNA and DNA could reverse protein deficiency that causes Friedreich's ataxia

Synthetic RNA and DNA could reverse protein deficiency that causes Friedreich's ataxia

'Housekeeping' gene may have a link to male infertility

'Housekeeping' gene may have a link to male infertility

Alternative splicing: a new approach to drug development? An interview with Lucy Donaldson

Alternative splicing: a new approach to drug development? An interview with Lucy Donaldson

FDA grants Orphan Drug Designation to Ionis Pharmaceuticals' IONIS-HTT Rx for treatment of HD patients

FDA grants Orphan Drug Designation to Ionis Pharmaceuticals' IONIS-HTT Rx for treatment of HD patients

Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

Isis Pharmaceuticals begins ISIS-FXI Rx Phase 2 study in patients with ESRD on hemodialysis

Isis Pharmaceuticals begins ISIS-FXI Rx Phase 2 study in patients with ESRD on hemodialysis

Genetic carrier screening: an interview with Don Hardison, CEO of Good Start Genetics

Genetic carrier screening: an interview with Don Hardison, CEO of Good Start Genetics

Clinical data from spinal muscular atrophy program presented at 20th International WMS Congress

Clinical data from spinal muscular atrophy program presented at 20th International WMS Congress

HSCI researchers find surprising similarities between SMA and ALS

HSCI researchers find surprising similarities between SMA and ALS

Determining the dynein-dynactin complex structure: an interview with Dr Gabriel C. Lander

Determining the dynein-dynactin complex structure: an interview with Dr Gabriel C. Lander

Researchers report role of two gene-regulating molecules in Parkinson's disease

Researchers report role of two gene-regulating molecules in Parkinson's disease

Isis Pharmaceuticals, AstraZeneca to develop antisense therapies for cardiovascular, metabolic, renal diseases

Isis Pharmaceuticals, AstraZeneca to develop antisense therapies for cardiovascular, metabolic, renal diseases

NEJM publishes positive clinical results from Phase 2 clinical study of volanesorsen

NEJM publishes positive clinical results from Phase 2 clinical study of volanesorsen