Frederic Revah

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gene therapy rare genetic disease clinical trials biotechnology research rare diseases neuromuscular diseases gene editing immunology bioproduction infant spinal muscular atrophy duchenne muscular dystrophy myotubular myopathy limb girdle muscular dystrophies wiskott-aldrich syndrome fanconis anemia crigler-najjar syndrome pompe disease leber hereditary optic neuropathy dna bank genetic research therapeutic strategies vector delivery clinical development research teams advanced technology platforms imaging cytometry preclinical assessment patient access gene therapy pipelines genetic defects therapeutic innovations biocluster artificial intelligence biomedicine cell & gene therapy patient-centric solution gene transfer genetic disorders long-term efficacy gene therapy trials scientific collaboration therapeutic solutions genome mapping therapeutic projects genetic therapies neuromuscular research innovative treatments research & development biotherapeutics gene therapy platforms biotechnology solutions clinical outcomes immune system disorders ocular diseases metabolic disorders preclinical research non-profit organization biotechnology pharmaceutical industry patient advocacy biomanufacturing gmp biomanufacturing dna & cell bank therapeutic research collaborations healthcare solutions pediatric patients ultra-rare conditions patient-centric outcomes therapeutic approaches genetic disease bank public donations afm-telethon global patient population healthcare providers disease focus treatment accessibility health care health wellness & fitness hospital & health care information technology & services