Leaders in pediatric neuromuscular research

Zaidman serves as the principal investigator on more than 15 clinical research trials in pediatric neuromuscular disorders. He supervises a team of six researchers who are helping develop innovative treatments. Based on experience in these clinical trials, we are aggressive and early adopters of the latest advanced therapies for neuromuscular disorders, including gene modifying treatments for spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).  


Our team members have been key participants in researching and developing innovative therapeutics for neuromuscular disorders that have achieved FDA approval:

Zolgensma

  • past participation in STR1VE trial for symptomatic SMA type 1 patients to receive IV zolgensma, STRONG trial for sitting but non-ambulatory SMA patient to receive IT zolgensma, SPR1NT trial for presymptomatic infants younger than 6 weeks with 2 or 3 copies for the SMN2 gene to receive IV zolgensma
  •  current participation in ongoing SMART trial for IV zolgensma in children with SMA up to 21kg, long term follow-up study for those in prior zolgensma clinical trials, RESTORE registry to follow newly diagnosed individuals with SMA receiving routine clinical care

Spinraza

  • past participation in CHERISH trial to assess nusinersen dosing in participants with later onset SMA, ENDEAR trial to assess nusinersen dosing in infants with SMA type 1 , and Expanded Access Program
  • current participation in SHINE trial for participants with SMA who previously participated in nusinersen studies to assess continued dosing, RESPOND trial to study nusinersen in participants with SMA who previously received Zolgensma, Spinraza in Adults with SMA (SAS) study to observe affect of adults with SMA type II/III initiating clinical treatment with Spinraza

Risdiplam

  • past participation in Expanded Access Program

Exondys 51

  • past participation in 4658-201 trial to evaluate Exondys 51 at 2 doses in boys with DMD, 4658-202 trial to evaluation ongoing efficacy and safety of continued dosing of Exondys 51 in boys with DMD who completed the 201 trial, 4658-203 to evaluate Exondys 51 administration in young males with early stage DMD, and 4658-204 study to evaluate safety and efficacy of Exondys 51 in participants with advanced stage DMD
  • Current participation in 4658-403 EVOLVE registry, an observational study for patients clinically receiving Exondys 51, Amondys 45, and Vyondys 53

Viltolarsen

  • Past participation in NS-065/NCNP-01-201 trial to assess safety and dosing of viltolarsen and NS-065/NCNP-01-202 trial, an extension study for boys with DMD who completed the 201 trial to continue viltolarsen and investigate ongoing safety and efficacy of dosing

Amondys 45 and Vyondys 53

  • Past participation in the 4045-301 ESSENCE trial, evaluating efficacy of Amondys 45 and Vyondys 53 compared to placebo in boys with DMD and the 4045-302 trial, a long term open label extension of the ESSENCE trial evaluating Amondys 45 and Vyondys 53
  • Current participation in 4658-403 EVOLVE registry, an observational study for patients clinically receiving Exondys 51, Amondys 45, and Vyondys 53

Lumizyme

  • Past participation in AGLU02704 study, a placebo-controlled stydy of safety and efficacy of Myozyme in patients with late-onset Pompe disease

We are currently participating in several clinical research trials for children with neuromuscular disorders. View our current clinical trials.

For more information on current trials, please email us at neuromusclepediatricresearch@wustl.edu.