ProQR Therapeutics released positive results from its phase 1/2 Stellar trial investigating QR-421a in patients with Usher syndrome and non-syndromic retinitis pigmentosa, according to a press release.
The therapy was well tolerated at all doses, and patients did not have any serious adverse events or inflammation, the release said.
Stellar, a randomized, sham-controlled, single ascending dose, global, multicenter study, included 20 subjects, 14 who received a single dose of QR-421a and six who received a single sham procedure for masking. Patients were organized into advanced and
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