Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Risdiplam: First Approval.

Drugs - 1 Nov 2020

Dhillon Sohita

Abstract excerpt

Risdiplam (Evrysdi™) is an orally administered, survival motor neuron 2 (SMN2)-directed RNA splicing modifier being developed by Roche, PTC Therapeutics Inc and the SMA Foundation for the treatment of the spinal muscular atrophy. The small molecule is designed to treat spinal muscular atrophy caused by mutations in chromosome 5q leading to SMN protein deficiency. The drug boosts the ability of an alternative gene...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Azo Compounds
Share to Clinical Trials, Phase II as Topic
Share to Clinical Trials, Phase III as Topic

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Drug Approval

Share to Multicenter Studies as Topic

Share to Muscular Atrophy, Spinal

Share to Survival of Motor Neuron 2 Protein

Share to United States Food and Drug Administration

Source

Identifiers

PMID: 33044711
DOI: 10.1007/s40265-020-01410-z

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Risdiplam: First Approval.PMID 33044711Read ↗

Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA)Semantic proximity Read ↗

Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in GermanySemantic proximity Read ↗

Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular AtrophySemantic proximity Read ↗

Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy TherapySemantic proximity Read ↗

The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular AtrophySemantic proximity Read ↗

Effectiveness and Safety of Risdiplam in Chinese Adolescents and Adults with Spinal Muscular Atrophy: A Prospective Real-World StudySemantic proximity Read ↗

An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular AtrophySemantic proximity Read ↗

Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent LandscapeSemantic proximity Read ↗

New Directions for SMA TherapySemantic proximity Read ↗

Disease Modifying Therapies for the Management of Children with Spinal Muscular Atrophy (5q SMA): An Update on the Emerging Evidence.Semantic proximity Read ↗

Advances in Therapeutic Development for Spinal Muscular AtrophySemantic proximity Read ↗

New treatments in spinal muscular atrophy.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.