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Trofinetide for the treatment of Rett syndrome: Long-term safety and efficacy results of the 32-month, open-label LILAC-2 study.

Autor: Percy AK; Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL, USA., Neul JL; Department of Pediatrics, Vanderbilt Kennedy Center, Vanderbilt University Medical Center, Nashville, TN, USA., Benke TA; Department of Pediatrics, Children's Hospital of Colorado/University of Colorado School of Medicine, Aurora, CO, USA., Berry-Kravis EM; Departments of Pediatrics, Neurological Sciences and Biochemistry, Rush University Medical Center, Chicago, IL, USA., Glaze DG; Department of Pediatrics and Neurology, Texas Children's Hospital/Baylor College of Medicine, Houston, TX, USA., Marsh ED; Division of Child Neurology, Children's Hospital of Philadelphia, Departments of Neurology and Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Barrett AM; Patient-Centered Outcomes Assessment, RTI Health Solutions, Research Triangle Park, NC, USA., An D; Acadia Pharmaceuticals, Inc., San Diego, CA, USA., Bishop KM; Acadia Pharmaceuticals, Inc., San Diego, CA, USA., Youakim JM; Acadia Pharmaceuticals, Inc., San Diego, CA, USA. Electronic address: jyouakim@acadia-pharm.com.

Publikováno v: Med (New York, N.Y.) [Med] 2024 Oct 11; Vol. 5 (10), pp. 1275-1281.e2. Date of Electronic Publication: 2024 Jul 17.

Effect of Hepatic Impairment on Trofinetide Exposures Using an In Silico Physiologically Based Pharmacokinetic Model.

Autor: Darwish M; Acadia Pharmaceuticals Inc., 12830 El Camino Real, Suite 400, San Diego, CA, 92130, USA. mdarwish@acadia-pharm.com., Obianom ON; Simulations Plus Inc., Buffalo, NY, USA., Youakim JM; Acadia Pharmaceuticals Inc., 12830 El Camino Real, Suite 400, San Diego, CA, 92130, USA., Darling I; Simulations Plus Inc., Buffalo, NY, USA., Lukacova V; Simulations Plus, Inc., Lancaster, CA, USA., Bradley H; Acadia Pharmaceuticals Inc., 12830 El Camino Real, Suite 400, San Diego, CA, 92130, USA.

Publikováno v: Advances in therapy [Adv Ther] 2024 Aug; Vol. 41 (8), pp. 3328-3341. Date of Electronic Publication: 2024 Jul 04.

Trofinetide Treatment Demonstrates a Benefit Over Placebo for the Ability to Communicate in Rett Syndrome.

Autor: Neul JL; Vanderbilt Kennedy Center, Vanderbilt University Medical Center, Nashville, Tennessee., Percy AK; University of Alabama at Birmingham, Birmingham, Alabama., Benke TA; Children's Hospital of Colorado, University of Colorado School of Medicine, Aurora, Colorado., Berry-Kravis EM; Rush University Medical Center, Chicago, Illinois., Glaze DG; Texas Children's Hospital, Baylor College of Medicine, Houston, Texas., Peters SU; Vanderbilt Kennedy Center, Vanderbilt University Medical Center, Nashville, Tennessee., Marsh ED; Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania., An D; Acadia Pharmaceuticals Inc, San Diego, California., Bishop KM; Acadia Pharmaceuticals Inc, San Diego, California., Youakim JM; Acadia Pharmaceuticals Inc, San Diego, California. Electronic address: jyouakim@ACADIA-Pharm.com.

Publikováno v: Pediatric neurology [Pediatr Neurol] 2024 Mar; Vol. 152, pp. 63-72. Date of Electronic Publication: 2023 Nov 23.