Diseases, Conditions, Syndromes
Study could aid degenerative disease therapies
Fresh insights into how nerves connect with muscles in the body could aid the development of therapies to treat neurodegenerative diseases.
Oct 30, 2019
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News tagged with spinal muscular atrophy
Genetics
Researchers create new protocol to improve gene therapy tool production
A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers.
Sep 19, 2019
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Medications
Lifesaving drug for infants costs $2.1 million a dose
(HealthDay)—Its extremely high price tag means that a lifesaving medication to treat young children with spinal muscular atrophy is simply too expensive for most families.
Jul 30, 2019
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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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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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