Patricia Inácio, PhD, science writer —

Patricia holds her PhD in cell biology from the University Nova de Lisboa, Portugal, and has served as an author on several research projects and fellowships, as well as major grant applications for European agencies. She also served as a PhD student research assistant in the Department of Microbiology & Immunology, Columbia University, New York, for which she was awarded a Luso-American Development Foundation (FLAD) fellowship.

Articles by Patricia Inácio

DYNE-101 receives orphan drug status for DM1 in Europe

Dyne Therapeutics’ investigational therapy DYNE-101 has been granted an orphan drug designation by the European Medicines Agency (EMA) for myotonic dystrophy type 1 (DM1). The designation is meant to support the development of potential therapies for rare, life-threatening, or chronically debilitating diseases. The benefits include reduced fees, clinical…

FDA puts AOC 1044 on fast track as Duchenne therapy

AOC 1044, an investigational exon 44-skipping therapy for Duchenne muscular dystrophy (DMD), has been granted fast track status by the U.S. Food and Drug Administration (FDA). The designation is intended to accelerate the therapy’s development and expedite its approval by providing more frequent meetings with the FDA and discussions…

FDA clears Phase 2 study of exon skipping therapy for DMD

Having reached an agreement with the U.S. Food and Drug Administration (FDA), NS Pharma will be launching a Phase 2 clinical trial to evaluate its investigational exon 44 skipping therapy for Duchenne muscular dystrophy (DMD). Trial details will be forthcoming once enrollment is imminent, according to NS Pharma,…

AOC 1020 wins FDA’s orphan drug status for treating FSHD

AOC 1020, Avidity Biosciences’ investigational therapy for facioscapulohumeral muscular dystrophy (FSHD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The designation is intended to encourage the development of therapies for rare diseases, those affecting fewer than 200,000 people in the U.S. It provides…

Potential DM1 Therapy, PGN-EDODM1, Shows Safety in Studies

PGN-EDODM1, a therapy candidate for myotonic dystrophy type 1 (DM1), showed a favorable safety profile in preclinical studies, including those in non-human primates, PepGen, the company developing the treatment, announced. This work is expected to support a request to initiate a single ascending dose clinical trial of PGN-EDODM1…