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Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification

Annals of clinical and translational neurology, 2023-11, Vol.10 (11), p.2155-2160

2023

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Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy

Muscle & nerve, 2021-10, Vol.64 (4), p.413-427

2021

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Onasemnogene abeparvovec for the treatment of spinal muscular atrophy

Expert opinion on biological therapy, 2022-09, Vol.22 (9), p.1075-1090

2022

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The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID‐19 pandemic

Muscle & nerve, 2020-07, Vol.62 (1), p.41-45

2020

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50 Years Ago in The Journal of Pediatrics: Pseudohypertrophic Muscular Dystrophy: Duchenne Type

The Journal of pediatrics, 2015-09, Vol.167 (3), p.620-620

Proud, Crystal M

2015

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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP‐9001‐103 (ENDEAVOR)

Annals of neurology, 2023-11, Vol.94 (5), p.955-968

2023

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Equitable Access of Delandistrogene Moxeparvovec for Patients With Duchenne Muscular Dystrophy: A Call for Discussion

Pediatric neurology, 2024-10, Vol.159, p.33-34

2024

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50 years ago in the Journal of Pediatrics: the use of serum creatine phosphokinase and other serum enzymes in the diagnosis of progressive muscular dystrophy

The Journal of pediatrics, 2013-12, Vol.163 (6), p.1656-1656

Proud, Crystal M

2013

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Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources

Advances in therapy, 2023-03, Vol.40 (3), p.1129-1140

2023

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50 years ago in the Journal of Pediatrics: Photogenic and self-induced epilepsy

The Journal of pediatrics, 2012-11, Vol.161 (5), p.874-874

Proud, Crystal M

2012

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

Journal of neuromuscular diseases, 2024-04, Vol.11 (3), p.687-699

2024

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Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations

Neurology. Clinical practice, 2025-02, Vol.15 (1)

2025

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Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

Journal of neuromuscular diseases, 2024-01, Vol.11 (2), p.425-442

2024

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Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

Journal of neuromuscular diseases, 2022-01, Vol.9 (5), p.655-660

2022

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50 Years Ago in T J P

The Journal of pediatrics, 2015-09, Vol.167 (3), p.620

Proud, Crystal M.

2015

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50 Years Ago in TheJournal ofPediatrics

The Journal of pediatrics, 2015-09, Vol.167 (3), p.620-620

Proud, Crystal M.

2015

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50 Years Ago in The Journal of Pediatrics

The Journal of pediatrics, 2013-12, Vol.163 (6), p.1656-1656

Proud, Crystal M.

2013

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50 Years Ago in The Journal of Pediatrics

The Journal of pediatrics, 2012-11, Vol.161 (5), p.874-874

Proud, Crystal M.

2012

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50 Years Ago in T he J ournal of P ediatrics

The Journal of pediatrics, 2015, Vol.167 (3), p.620-620

Proud, Crystal M., MD

2015

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Real-World Patterns of Nusinersen Use in Adult Patients with Spinal Muscular Atrophy (SMA) in the US: A Multi-Site Chart Review Study (2424)

Neurology, 2021-04, Vol.96 (15_supplement)

2021

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Six Month Results of Spinraza in Adults with Spinal Muscular Atrophy (SAS) Study (2446)

Neurology, 2021-04, Vol.96 (15_supplement)

2021

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50 Years Ago in T he J ournal of P ediatrics

The Journal of pediatrics, 2013, Vol.163 (6), p.1656-1656

Proud, Crystal M., MD

2013

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50 Years Ago in T he J ournal of P ediatrics

The Journal of pediatrics, 2012, Vol.161 (5), p.874-874

Proud, Crystal M., MD

2012

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Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians

Annals of the Child Neurology Society, 2024-09, Vol.2 (3), p.184-188

2024

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Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy

Pediatric neurology, 2024-04, Vol.153, p.11-18

2024

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Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification

Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy

Onasemnogene abeparvovec for the treatment of spinal muscular atrophy

The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID‐19 pandemic

50 Years Ago in The Journal of Pediatrics: Pseudohypertrophic Muscular Dystrophy: Duchenne Type

Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP‐9001‐103 (ENDEAVOR)

Equitable Access of Delandistrogene Moxeparvovec for Patients With Duchenne Muscular Dystrophy: A Call for Discussion

50 years ago in the Journal of Pediatrics: the use of serum creatine phosphokinase and other serum enzymes in the diagnosis of progressive muscular dystrophy

Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources

50 years ago in the Journal of Pediatrics: Photogenic and self-induced epilepsy

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

Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

50 Years Ago in T J P

50 Years Ago in TheJournal ofPediatrics

50 Years Ago in The Journal of Pediatrics

50 Years Ago in The Journal of Pediatrics

50 Years Ago in T he J ournal of P ediatrics

Real-World Patterns of Nusinersen Use in Adult Patients with Spinal Muscular Atrophy (SMA) in the US: A Multi-Site Chart Review Study (2424)

Six Month Results of Spinraza in Adults with Spinal Muscular Atrophy (SAS) Study (2446)

50 Years Ago in T he J ournal of P ediatrics

50 Years Ago in T he J ournal of P ediatrics

Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians

Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy

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