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

Autor: Percy, Alan K., Neul, Jeffrey L., Benke, Timothy A., Berry-Kravis, Elizabeth M., Glaze, Daniel G., Marsh, Eric D., Barrett, Amy M., An, Di, Bishop, Kathie M., Youakim, James M.

Publikováno v: In Med 11 October 2024 5(10):1275-1281

Burden of illness in Rett syndrome: initial evaluation of a disorder-specific caregiver survey.

Autor: Kaufmann, Walter E.^1,2 (AUTHOR) walter.e.kaufmann@emory.edu, Percy, Alan K.³ (AUTHOR), Neul, Jeffrey L.⁴ (AUTHOR), Downs, Jenny⁵ (AUTHOR), Leonard, Helen⁵ (AUTHOR), Nues, Paige⁶ (AUTHOR), Sharma, Girish D.⁷ (AUTHOR), Bartolotta, Theresa E.⁸ (AUTHOR), Townend, Gillian S.⁹ (AUTHOR), Curfs, Leopold M. G.⁹ (AUTHOR), Mariotti, Orietta¹⁰ (AUTHOR), Buda, Claude¹¹ (AUTHOR), O'Leary, Heather M.¹ (AUTHOR), Oberman, Lindsay M.¹² (AUTHOR), Vogel-Farley, Vanessa¹ (AUTHOR), Barnes, Katherine V.¹ (AUTHOR), Missling, Christopher U.¹ (AUTHOR)

Publikováno v: Orphanet Journal of Rare Diseases. 8/13/2024, Vol. 19 Issue 1, p1-14. 14p.

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

Autor: Neul, Jeffrey L., Percy, Alan K., Benke, Timothy A., Berry-Kravis, Elizabeth M., Glaze, Daniel G., Peters, Sarika U., Marsh, Eric D., An, Di, Bishop, Kathie M., Youakim, James M.

Publikováno v: In Pediatric Neurology March 2024 152:63-72

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