Genetics
The gene therapy revolution is here
Gene therapy—for so long something that belonged to the future—has just hit the streets.
Jun 05, 2019
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News tagged with spinal muscular atrophy
Medications
FDA approves first gene therapy for spinal muscular atrophy
(HealthDay)—The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy (SMA), the U.S. Food and Drug Administration announced Friday.
May 28, 2019
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Medications
FDA approves $2M medicine, most expensive ever
U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby's muscle control and kills nearly all of those with the most common type of the disease within a couple of years.
May 24, 2019
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Neuroscience
Spinal muscular atrophy drug may help kids with later-onset disease
There is now further evidence that a drug that is effective in treating the rare muscle-wasting disease spinal muscular atrophy (SMA) early in life may be associated with improvement in older children, according to a study ...
Apr 24, 2019
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Medical research
Spinal organoids mimic neurodegenerative disease
'Organoids' that mimic the developing spinal cord could assist research and drug development for neurodegenerative diseases such as spinal muscular atrophy and amyotrophic lateral sclerosis.
Mar 18, 2019
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Neuroscience
Researchers 'bait' pathological proteins underlying many neurodegenerative disorders
A single misbehaving protein—called TDP-43—is behind 97 percent of amyotrophic lateral sclerosis (ALS) cases and 45 percent of frontotemporal dementia diagnoses. It also is found in 80 percent of chronic traumatic encephalopathy ...
Feb 27, 2019
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Genetics
New study offers hope for patients suffering from a rare form of blindness
A new form of therapy may halt or even reverse a form of progressive vision loss that, until now, has inevitably led to blindness. This hyper-targeted approach offers hope to individuals living with spinocerebellar ataxia ...
Nov 01, 2018
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Genetics
Researchers correlate spinal muscular atrophy disease expression with haplotypes
A natural history study has provided the first comprehensive clinical description of spinal muscular atrophy (SMA) within the Amish and Mennonite communities and correlates ancestral chromosome 5 haplotypes and SMN2 copy ...
Sep 06, 2018
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Neuroscience
Spinal muscular atrophy drug may be effective if started later than previously shown
A drug shown to be effective in the treatment of babies with the rare muscle-wasting disease spinal muscular atrophy may be effective for muscle control even when treatment is started in children seven months and older, according ...
Aug 29, 2018
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Neuroscience
New nusinersen drug delivery method identified for spinal muscular atrophy patients
STRASBURG, PA- A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy (SMA) using a subcutaneous intrathecal catheter system (SIC) configured by connecting an intrathecal ...
Aug 24, 2018
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