Tag: Muscular Atrophy

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German researchers recommend early screening and treatment for spinal muscular atrophy

Newsemia
The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children...
Neurology

Researchers identify a natural amino acid as potential treatment for polyQ diseases

Newsemia
Familial spinocerebellar ataxia (SCA), Huntington disease, and spinal and bulbar muscular atrophy are inherited neurodegenerative diseases. Source link...
Neurology

Maybe Memorizing the Krebs Cycle Was Worthwhile After All

Newsemia
Mitochondrial disorders are often difficult to diagnose and treat due to their multisystem nature. As scientists better understand mitochondrial function, effective treatments are beginning to...
Neurology

The Falls and Frustrations of Progressive Supranuclear Palsy

Newsemia
PSP is a rare neurogenerative disorder that is increasingly appreciated among neurologists. Yet effective treatments remain elusive. Medscape Neurology Source link...
Neurology

SUNFISH: Risdiplam Meets Primary Endpoint in SMA

Newsemia
Risdiplam, an oral investigational agent designed to increase and sustain SMN protein levels of patients with spinal muscular atrophy (SMA), met the primary endpoint in...
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Faster, cheaper, better: improved gene therapy tool production

Newsemia
The heart of modern therapy is increasingly personalized and targeted at specific cellular or genetic deficits. This requires gene therapy, in many cases. A new...
Latest News

Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

Newsemia
James Wilson, MD, PhD, recalls being struck by the devastating toll of rare diseases as a young physician in the 1980s. He set out on...
Neurology

What's Hot at AAN 2019?

Newsemia
‘Philly Spectacular’ is the theme of this year’s AAN Annual Meeting, says the event’s science committee chair, adding that more than 15,000 attendees are expected...
Latest News

Unique gene therapy approach paves new way to tackle rare, inherited diseases

Newsemia
Nonsense mutations are single-letter errors in the genetic code that prematurely halt the production of critical proteins. These unfinished proteins are unable to function normally,...
Neurology

Spasticity in MND Improved With Cannabinoid Oral Spray

Newsemia
Nabiximols, a cannabinoid-based oral spray approved outside the US for MS symptoms, shows improvement of spasticity in amyotrophic and primary lateral sclerosis when used as...
Neurology

FDA OKs First Treatment for Lambert-Eaton Myasthenic Syndrome

Newsemia
Amifampridine (Firdapse) is approved for adults with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder characterized by muscle weakness. FDA Approvals Source link...
Latest News

Alternative method identified for delivering nusinersen to spinal muscular atrophy patients

Newsemia
A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy using a subcutaneous intrathecal catheter system configured by...

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