Avidity Biosciences (RNA) unveiled promising results for its Duchenne muscular dystrophy treatment on Friday. .
Patients with Duchenne muscular dystrophy, or DMD, don't make enough dystrophin. The dystrophin protein helps keep muscles intact. After receiving Avidity's drug for four months, patients had a 25% increase in dystrophy production and reached dystrophin levels that were at 54% of normal.
Chardan analyst Keay Nakae lists RNA stock as a top pick for 2024. He says the results help validate Avidity's approach. The drug, del-zota, works by skipping a genetic mutation called exon 44 to boost the production of dystrophin.
"This data positions the company to continue to evaluate additional DMD 44 patients in order to accumulate sufficient patient experience that could support a possible accelerated approval," Nakae said in a report to clients.
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