Sankarasubramon Subramony

CMS Newborn Screening - COQHX

Motor Outcomes to Validate Evaluations in FSHD (MOVE) Plus MRI and Biomarker Discovery/Validation (MOVE+)

Care Center type Adult MDA clinic, University of Florida

A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1

An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects with Myotonic Dystrophy Type I

A Phase 1/Phase 2 Open-Label Single Arm Study With Dose Escalation (Part A), and Dose Expansion (Part B) Parts to Evaluate the Safety, Tolerability, and Efficacy of SAR446268, An Adeno-Associated Viral Vector-Mediated Gene Therapy In Participants 10 To 50 Years Old With Non-Congenital Myotonic Dystrophy Type 1

A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled Study (Part 1) and Open-Label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich’s Ataxia Aged 2 to < 16 Years

A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

wearable device cohort of the MOVE+ Substudy (the “MOVE Study”, including a substudy, the “MOVE+ Substudy”, collectively, the “Study”) under the protocol entitled “Motor Outcomes to Validate Evaluations in FSHD (MOVE) Plus MRI and Biomarker Discovery/Validation (MOVE+)” (the “Protocol”);

A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (FREEDOM2-DM1)

296FA401 (408-C-2301) - An observational, multinational, post-marketing registry of omaveloxolone treated patient (“PAS Study”).

Avidity Biosciences Research Asst. Support

A Phase 1/2, Randomized, Double-blind, Placebo controlled Single Dose and Multiple dose Escalation Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of VX 670 in Adult Subjects with Myotonic Dystrophy Type 1 VX23-670-001

A Phase 3 Randomized, Double-Blind, Placebo Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type

A Phase 3 Randomized, Double-Blind, Placebo Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type

AOC 1020-CS2, A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, and Pharmacokinetics of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

The MDA Care Center at University of Florida Health: FY25-FY27

A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Clinical Efficacy of Intravenous Administration of ATX-01 In Male and Female Participants aged 18 to 64 with Classic Myotonic Dystrophy Type 1 (DM1)

END-DM1 Patient Reimbursement

Jive: An Open Label Extension Study to Assess the Longterm Safety, Efficacy, Pharmacodynamics, Pharmacokinetics, and Tolerability of Subcutaneous CTI-1601 in Subjects with Friedreich’s Ataxia

Trial Readiness and Endpoint Assessment in LGMDR1 (TREATing-LGMDR1)

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study #1016

Circadian Rhythm Disturbances in Myotonic Dystrophy Type 1

Enhancing Our Understanding of Physical Activity in Myotonic Dystrophy Type 1