Gene therapy medication provides improvements in severe SMA
Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of the spinal muscular atrophy...
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Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of the spinal muscular atrophy...
Drugs normally used to treat cancer could reduce the disfigurements of thousands of children born with life-threatening blood vessel defects...
Amglidia is a new formulation of glibenclamide, specifically developed for use in newborns, toddlers and children...
A preliminary Phase 2 clinical trial has demonstrated that patients with acute ischemic stroke, can safely tolerate high doses of 3K3A-APC, a promising anti-stroke drug...
An investigational compound designed to block a hyperactive cell growth signal in advanced melanoma and other cancers has shown some promise in an early-stage clinical trial...
Alzheimer's drug appears to reverse structural and genetic brain changes that affect memory and learning...
An antibody used to treat the skin disease psoriasis is also effective at reducing aortic inflammation, a key marker of future risk of major cardiovascular events...
Novartis and the Bill & Melinda Gates Foundation have formed an alliance to advance the development of Novartis' drug candidate KDU731...
The FDA has approved a label update for Cosentyx, the first interleukin-17A inhibitor approved to treat moderate-to-severe plaque psoriasis...
Researchers have shown that two different compounds can safely and effectively be added to treatment regimens to block the transmission of the most common form of malaria...
A randomised, double-blind, placebo-controlled trial has found that sodium benzoate improves symptoms in clozapine-resistant schizophrenia...
Scientists in the United States have discovered that two existing FDA-approved drugs, clofarabine (Clolar™) and cladribine (Leustatin™), have a powerful inhibitory impact on Ewing sarcoma cells in the lab.
A drug used to treat seizures may effectively treat the movement symptoms in people with dementia with Lewy bodies without causing additional psychiatric symptoms...
A team of UK scientists have harnessed an innovative new method to gain a greater understanding of signalling stations within living cells, called nanodomains.
Replacing daily pills with a weekly regimen could help patients stick to their dosing schedule...