Jerry R. Mendell

Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

Cardiac and pulmonary findings in dysferlinopathy: A 3‐year, longitudinal study

Water T2 could predict functional decline in patients with dysferlinopathy

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial

Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue

Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1

Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale

Current Clinical Applications of In Vivo Gene Therapy with AAVs

Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes

Gene-Replacement Therapy in Spinal Muscular Atrophy Type 1: Long-Term Follow-Up From the Onasemnogene Abeparvovec-xioi Phase 1/2a Clinical Trial (1808)

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials

Examining longitudinal functional changes in Dysferlinopathy: The JAIN Clinical Outcome Study (P5.429)

Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice

MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin

The Clinical Outcome Study for dysferlinopathy

Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy

Clinical phenotypes as predictors of the outcome of skipping around <scp><i>DMD</i></scp> exon 45

VIP-expressing Dendritic Cells Protect Against Spontaneous Autoimmune Peripheral Polyneuropathy

Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases

Evidence‐based path to newborn screening for duchenne muscular dystrophy

Essential metabolic, anti-inflammatory, and anti-tumorigenic functions of miR-122 in liver

<i><scp>LTBP4</scp></i> genotype predicts age of ambulatory loss in duchenne muscular dystrophy