Data from first adult patient dosed in REVEAL Phase 1/2 trial showed TSHA-102 was well-tolerated with no treatment-emergent serious adverse events (SAEs) as of six-week assessment and improvement in ...

Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today provided an update on ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has granted marketing authorization of DAYBUE™ (trofinetide) for the treatment of Rett ...

Available clinical data from the two adult patients dosed with TSHA-102 in the first cohort (low dose) to be discussed during upcoming quarterly earnings call following Independent Data Monitoring ...

SAN DIEGO--(BUSINESS WIRE)-- Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA ...

Natural history data analysis established patients ≥ six years of age are in developmental plateau, with a ~0% likelihood of gaining/regaining developmental milestones across the core functional ...

Health Canada authorized the Company’s protocol amendment that expands eligibility to include patients aged 12 and older with stage four Rett syndrome in the REVEAL Phase 1/2 adult trial in Canada ...

MONEY AND AWARENESS. CONCORD HIGH SCHOOL STUDENT IZZY BOOM IS BUSY WITH HER FRIENDS EMMA AND SAMMY. OH YEAH, I THINK THIS ONE’S MY FAVORITE DECORATING DUCKS. OH, THAT’S A CUTE ONE. TWO, TWO DUCK. BUT ...

For TSHA-102 in Rett syndrome, we believe the initial safety profile and significant clinical improvements seen in the first adult patient with severe disease four weeks post-treatment reinforces the ...

--DAYBUE is the first and only therapy approved in Canada for the treatment of Rett syndrome, a rare, neurodevelopmental disorder SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) ...