HELIOS is a Phase II trial evaluating the safety, tolerability and effects of AMX0035 in adults with Wolfram syndrome.

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European regulators have for the fourth time issued a negative opinion on PTC's Translarna. Elsewhere, Amylyx read out data ...

过去几天对生物科技公司Amylyx Pharmaceuticals ...

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H.C. Wainwright维持对Amylyx Pharmaceuticals Inc. (NASDAQ: AMLX)的买入评级，目标价格保持在$8.00。 该公司的认可是在Amylyx最近宣布积极的试验结果之后。周四披露的HELIOS试验数据显示，主要终点C肽水平和次要生物标志物HbA1c水平均持续改善。这些结果表明，Wolfram综合征患者的疾病进展有所减缓。 延长至36周和48周的试验数据 ...