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1#First small-molecule drug targeting RNA gains momentum(来源:Nature Biotechnology) 2#盘点:2020年FDA批准的新药(来源:医药魔方) 3#The RNA drug hunters(来源:c & en) 4#Streptomycin(来源:Wikipedia) 5#Matthew G C , Jessica L , Childs-Disney, et al. How We Think about Targeting RNA with Sma...
Evrysdi, the first oral drug for spinal muscular atrophy, has shown encouraging results in a variety of patient groups with more systemic drug exposure. (Courtesy of Roche Korea) The first oral drug for spinal muscular atrophy (SMA), Evrysdi (risdiplam), has produced encouraging results in var...
Biohaven Pharmaceutical Holding announced the acquisition of Channel Bio and new licensing rights to a spinal muscle atrophy drug from Bristol Myers Squibb.
Drug therapiesThere has been huge development in SMA treatment options in the past decade.Three disease-modifying therapies are available for SMA. They all have been shown to help infants achieve otherwise unattainable motor milestones, and to improve or maintain motor, breathing, and swallowing ...
Repligen Corporation (NASD: RGEN) today announced positive results from a Phase 1 study to evaluate the pharmacokinetic (PK) and safety profile of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy (SMA). SMA is a inherited neurodegenerative disease...
This research represents the first longitudinal study that has monitored SMN levels in response to a drug that modulates gene expression. Learn more Valproic Acid Valproic acid has long been indicated for the treatment of seizures, mania, and migraine prophylaxis and has been used off-label for ...
The U.S. Food and Drug Administration (FDA) approved Evrysdi in August 2020, making it the country’s third new disease-modifying treatment for SMA in four years.It initially was approved for adults and children 2 months and older with all types of SMA; that indication was extended in ...
the company is developing small molecule drug candidates specifically engineered to impact muscle function and contractility. Cytokinetics is collaborating with Amgen Inc. (Amgen) to developomecamtiv mecarbil, a novel cardiac muscle activator.Omecamtiv mecarbilis the subject of an inter...