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Ionis Pharmaceuticals has dosed the first subject in the global, randomised Phase III REVEAL trial of investigational ION582.
That night, Young spent hours researching online and came across Angelman syndrome, sometimes referred to as "happy puppet" or "happy baby" syndrome because of the characteristic joyful demeanor ...
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The firm expects to complete enrolling the REVEAL trial, in which patients will receive the ASO, dubbed ION582, in 2026.
"Angelman syndrome is a complex neurodevelopmental ... standing up by themselves or taking their first steps. Infants can develop microcephaly, a condition where their heads are characteristically ...
“Dosing the first person with Angelman syndrome in our pivotal REVEAL study ... communication as assessed by the Bayley Scales for Infant and Toddler Development-4 (Bayley-4), an objective ...
After welcoming baby Huxley on August 22 ... called to their paediatrician in Broken Hill and told Huxley had Angelman syndrome, a rare genetic condition that affects the functioning of nerves ...
Angelman syndrome, which affects 1 in 15,000 people ... severe developmental delays and is typically first noticed when a baby is not crawling or babbling between 6 and 12 months, according ...
Angelman syndrome is a rare neurological condition ... “James was a … very silent baby … he didn’t make any sounds at all. I just thought I’d hit the lottery, you know, I thought I ...