Risdiplam (evrysdi) - Neonatal Disorders

Relevant Publications

Global Risdiplam Compassionate Use Program for Patients With Type 1 or 2 Spinal Muscular Atrophy.

Issue Release: 2024

[Risdiplam for the treatment of spinal muscular atrophy].

Issue Release: 2024

Spinal Muscular Atrophy Scoliosis in the Era of Background Therapies-A Review of the Literature.

Issue Release: 2024

Estimation of FMO3 Ontogeny by Mechanistic Population Pharmacokinetic Modelling of Risdiplam and Its Impact on Drug-Drug Interactions in Children.

Issue Release: 2023

An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy.

Issue Release: 2023

Current and emerging targeted therapies for spinal muscular atrophy.

Issue Release: 2023

Targeting strategies for modulating pre-mRNA splicing with small molecules: recent advances.

Issue Release: 2022

[Adult Spinal Muscular Atrophy].

Issue Release: 2022

Curing SMA: Are we there yet?

Issue Release: 2022

Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders.

Issue Release: 2022

Risdiplam: A Review in Spinal Muscular Atrophy.

Issue Release: 2022

RNA-based drug discovery for spinal muscular atrophy: a story of small molecules and antisense oligonucleotides.

Issue Release: 2022

In brief: Risdiplam (Evrysdi) for spinal muscular atrophy.

Issue Release: 2021

SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.

Issue Release: 2021

Model-Based Drug-Drug Interaction Extrapolation Strategy From Adults to Children: Risdiplam in Pediatric Patients With Spinal Muscular Atrophy.

Issue Release: 2021

Addressing Today\'s Absorption, Distribution, Metabolism, and Excretion (ADME) Challenges in the Translation of In Vitro ADME Characteristics to Humans: A Case Study of the mRNA Splicing Modifier Risdiplam.

Issue Release: 2021

Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment.

Issue Release: 2020

The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy.

Issue Release: 2020

Current and emerging therapies for Duchenne muscular dystrophy and spinal muscular atrophy.

Issue Release: 2020

Risdiplam: First Approval.

Issue Release: 2020

What is the Role of Nutrition in Child Development?

Why is funding important in Pediatrics?

Are There Risks Associated with Using Surgical Guides?

What are the challenges in user interface design?

What Are Some Common Misconceptions?

What is Sapropterin?

What Are the Risks of an Improperly Fitted Car Seat?

How Are Abnormal Immunoglobulin Levels Treated in Neonates?

How is Osmolality Managed in Neonates?

What Role Does Genetic Counseling Play?

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