180 Systemic delivery of factor IX messenger RNA for protein replacement therapy.

179 CD6 as a potential target for treating multiple sclerosis.

178 A nontoxic pain killer designed by modeling of pathological receptor conformations.

177 Pharmacologic inhibition of Hsp90 to prevent GLT-1 degradation as an effective therapy for epilepsy.

176 Allosteric “beta-blocker” isolated from a DNA-encoded small molecule library.

175 Drug discovery for Diamond-Blackfan anemia using reprogrammed hematopoietic progenitors.

174 Thioredoxin reverses age-related hypertension by chronically improving vascular redox and restoring eNOS function.

173 A Highly Durable RNAi Therapeutic Inhibitor of PCSK9.

172 SC83288 is a clinical development candidate for the treatment of severe malaria.

171 6’-Sialyllactose conjugated to polyamidoamine dendrimers at a well-defined valency and spacing can circumvent drug resistance and inhibit influenza A viruses.

170 Five-coordinate H64Q neuroglobin as a ligand-trap antidote for carbon monoxide poisoning.

169 Diversity-oriented synthesis yields novel multistage antimalarial inhibitors.

168 Purinergic receptors in the carotid body as a new drug target for controlling hypertension.

167 Efficacy and safety of benralizumab for patients with severe asthma uncontrolled with high-dosage inhaled corticosteroids and long-acting β2-agonists (SIROCCO).

166 Benralizumab, an anti-interleukin-5 receptor α monoclonal antibody, as add-on treatment for patients with severe, uncontrolled, eosinophilic asthma (CALIMA).

165 Fragment-based discovery of a new family of non-peptidic small-molecule cyclophilin inhibitors with potent antiviral activities.

164 Treatment of otitis media by transtympanic delivery of antibiotics.

163 A selective inhibitor of the kinetoplastid proteasome (GNF6702) is identified that is highly efficacious in vivo, clearing the parasites that cause leishmaniasis, Chagas disease and sleeping sickness from mice, highlighting the possibility of developing a single class of drugs for these neglected diseases.

162 Like citrate, the molecule hydroxycitrate is shown to inhibit growth of the crystal that is the principal component of kidney stones, suggesting that hydroxycitrate could be another treatment for kidney stone disease.

161 Cutaneous wound healing through paradoxical MAPK activation by BRAF inhibitors.

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