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.
CSHL researcher-inventor hails FDA approval of new SMA drug

CSHL researcher-inventor hails FDA approval of new SMA drug

Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

MDA celebrates FDA approval of new spinal muscular atrophy drug

MDA celebrates FDA approval of new spinal muscular atrophy drug

FDA approves first drug to treat children and adults with spinal muscular atrophy

FDA approves first drug to treat children and adults with spinal muscular atrophy

Advances in RNA research could pave new way for development of anti-cancer drugs

Advances in RNA research could pave new way for development of anti-cancer drugs

Research findings point to potential new targeted therapy for SMA

Research findings point to potential new targeted therapy for SMA

HMS study reveals key instigator of nerve cell damage in ALS patients

HMS study reveals key instigator of nerve cell damage in ALS patients

First investigational treatment for infantile-onset SMA shows promising results in clinical trial

First investigational treatment for infantile-onset SMA shows promising results in clinical trial

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