Selected Publications

Gavazzi F, Woidill S, Sevagamoorthy A, Jawad AF, D’Aiello R, Bradford J, Lerner M, Hong P, White A, Tashnim Z, Cusack SV, Glanzman AM, Harrington A, Waldman AT, Vanderver A, Adang L: Design of a pediatric low motor function item batter in leukodystrophies. Child Neurology Society Annual Meeting, Montreal, Quebec, Canada Oct 2026.

Yarlas A, Maher S, Llonch MV, Kymes S, Vilcot T, Collins A, Waldman A, on behalf of the ION373-CS1 Study Team.: Psychometric Evaluation of the 10-Meter Walk Test in Alexander Disease (AxD): Findings from the ION373-CS1 study. Child Neurology Society Annual Meeting, Montreal, Quebec, Canada Oct 2026.

Waldman AT, Tonduti D, van der Knaap MS, Bernard G, Eichler F, Sampson JB, Zerem A, Chataway J, Cunningham C, Bertini E, Nicita F, Hacohen Y, Saito T, Keller SR, Vaia Y, Kim NN, van Haren K, Nagy A, van den Berg E, Bruschi F, Lynch DS, Chen C, Ruiz-Santiago N, Yarlas A, Cowling BS, Collins A, Smith AV: Efficacy and safety of zilganersen, an investigational RNA-targeted antisense therapy, in people living with Alexander disease: additional data from the pivotal phase 1-3 trial. Child Neurology Society Annual Meeting, Montreal, Quebec, Canada Oct 2026.

Moore K, Bonkowsky JL, Keller S, Waldman AT, Junge C, Miller G, Wolf NI: Study Design of ROCKET and ORBIT and Initial Insights Into Disease Trajectory in Pelizaeus-Merzbacher Disease. Child Neurology Society Annual Meeting, Montreal, Quebec, Canada Oct 2026.

Kymes S, Roberts A, Waldman A, Williams K, Collins A, Bernarde J, Matter E, Savar S, Bernard G, Keller SR, Cunningham C, Eichler F, Hacochen Y, Sampson J, Currlin W, Hazel S, Lee L, Vera-Llonch1 M, for the ION373-CS1 research group: In-trial interviews to explore the experience of individuals with Alexander disease in the zilganersen phase 3 clinical trial. Child Neurology Society Annual Meeting, Montreal, Quebec, Canada Oct 2026.

Kymes S, Vera Llonch M, Chen C, Waldman A, Ruiz Santiago N, Collins A.: Reduction in frequency of all-cause hospitalizations from the ION373-CS1 clinical trial of zilganersen in the treatment of individuals with Alexander disease. Child Neurology Society Annual Meeting, Montreal, Quebec, Canada Oct 2026.

Marefi A, Grasso EA, Garber L, Narula S, Waldman A, Bar-Or A, Banwell B, Hopkins SE: Factors Influencing Early and Late B-Cell Repopulation After Rituximab Therapy in Pediatric Central Nervous System Inflammatory Disorders. Pediatr Neurol 180: 1-8, July 2026.

Bach AM, Chang G, Grasso EA, Hopkins S, Narula S, Waldman A, Banwell B: Association of Cerebrospinal Fluid-Restricted Oligoclonal Bands With Relapsing Pediatric Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease. Pediatr Neurol Page: Epub ahead of print, Jun 2026.

Chang G, Nasr Z, Schoeps VA, Virupakshaiah A, Zamvil SS, Moseley CE, Sabatino JJ Jr, Chong J, Graves JS, Waltz M, Rodriguez M, Tillema JM, Chitnis T, Rensel M, Abrams AW, Krupp L, Waldman AT, Mar S, Schreiner T, Rose J, Casper TC, Waubant E: Adenotonsillectomy is associated with increased risk and disease activity in pediatric-onset multiple sclerosis. Mult Scler Page: Epub ahead of print, May 2026.

Karandikar S, Sevagamoorthy A, Zimmerman D, D'Aiello R 3rd, Dorfschmidt L, Cyr K, Jung B, Levitis E, Adang LA, Arnold K, Bennett ML, Charsar BA, Dominguez Gonzalez CA, Gavazzi F, Hong P, Orthmann-Murphy JL, Pham ST, Kelley K, Lerner M, Shults J, Thakur N, Vossough A, Waldman AT, White A, Whitehead MT, Emrick L, Fraser J, Van Haren K, Keller S, Fatemi A, Eichler F, Bonkowsky JL; Global Leukodystrophy Initiative Clinical Trials Network Workgroup; Seidlitz J, Alexander-Bloch AF, Vanderver A: Normative modeling for quantitative brain MRI phenotyping and biomarker discovery for pediatric leukodystrophies. medRxiv [Preprint] May 2026.