Muscular Atrophy News and Research

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

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

Researchers identify new molecular targets for recovering motor function in men afflicted with SBMA

Researchers identify new molecular targets for recovering motor function in men afflicted with SBMA

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

UF Health investigators make new discovery about Huntington's disease

UF Health investigators make new discovery about Huntington's disease

MedUni Vienna researchers discover genetic cause of a rare disease

MedUni Vienna researchers discover genetic cause of a rare disease

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

Long-term disease progression in spinocerebellar ataxia quantified

Long-term disease progression in spinocerebellar ataxia quantified

Special issue of Future Science OA highlights research on protein misfolding diseases

Special issue of Future Science OA highlights research on protein misfolding diseases

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