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Results for “SMA1”

Showing 13 of 0 results

Not applicableUnknownNCT05475691
What this trial is testing

Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America

Who this might be right for
Spinal Muscular Atrophy
Hospital Israelita Albert Einstein 361
Testing effectiveness (Phase 2)Study completedNCT00568698
What this trial is testing

A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients

Who this might be right for
Muscular Atrophy, Spinal
Stanford University 29
Large-scale testing (Phase 3)Study completedNCT03837184
What this trial is testing

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

Who this might be right for
Spinal Muscular Atrophy Type I
Novartis Gene Therapies 2
Not applicableNot Yet RecruitingNCT07596277
What this trial is testing

Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication

Who this might be right for
Spinal Muscular Atrophy 1
Guy's and St Thomas' NHS Foundation Trust 15
Testing effectiveness (Phase 2)Enrolling By InvitationNCT05614531
What this trial is testing

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

Who this might be right for
Spinal Muscular Atrophy Type I
Guangzhou Jiayin Biotech Ltd 12
Testing effectiveness (Phase 2)Looking for participantsNCT07070999
What this trial is testing

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

Who this might be right for
Spinal Muscular Atrophy Type I
Gemma Biotherapeutics 22
Testing effectiveness (Phase 2)Study completedNCT00661453
What this trial is testing

CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

Who this might be right for
Spinal Muscular Atrophy Type I
University of Utah 40
Not applicableLooking for participantsNCT07332702
What this trial is testing

Long Read Analysis in Spinal Muscular Atrophy - LOREASI

Who this might be right for
Spinal Muscular Atrophy (SMA)
University Hospital, Rouen 27
Early research (Phase 1)Study completedNCT02122952
What this trial is testing

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

Who this might be right for
Spinal Muscular Atrophy 1
Novartis Gene Therapies 15
Not applicableUnknownNCT06256887
What this trial is testing

Sleep Spindles Organization as an Early Neural Marker of Neuromotor Outcome

Who this might be right for
EEG Sleep SpindlesMotor OutcomeCerebral Palsy+1 more
IRCCS Fondazione Stella Maris 80
Not applicableEnded earlyNCT02550691
What this trial is testing

Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene

Who this might be right for
Spinal Muscular Atrophy
University Hospital, Rouen 48
Not applicableStudy completedNCT06864767
What this trial is testing

Impact of Motor and Oral Motor Function on Quality of Life in Children With SMA

Who this might be right for
Spinal Muscular Atrophy Type I
Zeynep HOŞBAY 23
Not applicableStudy completedNCT03395795
What this trial is testing

Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II

Who this might be right for
Infantile Spinal Muscular Atrophy
Assistance Publique - Hôpitaux de Paris 1

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