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.
Positive CHMP opinion recognizes efficacy of Biogen’s SPINRAZA for treatment of spinal muscular atrophy

Positive CHMP opinion recognizes efficacy of Biogen’s SPINRAZA for treatment of spinal muscular atrophy

Targeted treatment slows progression of two degenerative brain diseases in mice

Targeted treatment slows progression of two degenerative brain diseases in mice

Researchers use mathematical modeling to raise density of bones badly degraded by osteoporosis

Researchers use mathematical modeling to raise density of bones badly degraded by osteoporosis

Emory researchers reveal 'matchmaker' role for protein linked to SMA

Emory researchers reveal 'matchmaker' role for protein linked to SMA

Designer compound may be effective tool for tackling tau-related disorders

Designer compound may be effective tool for tackling tau-related disorders

Synthetic molecule appears to reverse Alzheimer's-related neurological damage

Synthetic molecule appears to reverse Alzheimer's-related neurological damage

Orphan Disease Center establishes new initiative that focuses on ALS

Orphan Disease Center establishes new initiative that focuses on ALS

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

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

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