Glycomine has concluded subject enrolment in the Phase IIb POLAR study of GLM101 to treat phosphomannomutase 2 congenital ...

POLAR is a randomized, double-blind, placebo-controlled study that enrolled 43 patients with PMM2-CDG across 15 sites ...

Emily Felix, 28, and her family were ‘killed’ by the news that a drug to treat Friedreich’s ataxia may not be considered for ...

SUNRISE-FA 2 open-label, pivotal trial protocol and SAP for LX2006 submitted to FDA in Q1 2026 following Type B meeting; study initiation on ...

CHARLESTOWN, Mass., April 07, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for ...

Sanofi (NASDAQ:SNY) is one of the best undervalued stocks under $50 to invest in now. Sanofi (NASDAQ:SNY) announced on March 18 that the US Food and Drug Administration (FDA) has granted Breakthrough ...

She was diagnosed three years ago and her symptoms are likely to worsen over time, eventually leading to her dependence on a ...

Breakthrough Therapy Designation granted to nomlabofusp for the treatment of adults and children with FA based on FDA’s review of available clinical data from open label studyContinued alignment with ...

CEO Al Sandrock outlined several 2026 priorities in a discussion focused on the company’s tau-directed pipeline and its newer blood-brain barrier (BBB)-penetrant capsid platform. Sandrock described ...

- Strong Capital Position: Cash, cash equivalents and available-for-sale securities of $187.9 million at December 31, 2025; in March 2026, the Company completed a $240 million private placement; the ...

While recent FDA guidance speaks to the agency’s support of innovative trial designs—including the use of external ...