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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

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Risdiplam: A Review in Spinal Muscular Atrophy.

¹Springer Nature, Mairangi Bay, Private Bag 65901, Auckland, 0754, New Zealand. demail@springer.com.

|March 14, 2022

Summary

This summary is machine-generated.

Risdiplam is the first oral medication for spinal muscular atrophy (SMA). Clinical trials show it improves motor function in SMA patients and is well-tolerated.

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Area of Science:

Neurology
Genetics
Pharmacology

Background:

Spinal muscular atrophy (SMA) is a genetic disorder characterized by motor neuron loss.
The pathophysiology of SMA is driven by a lack of full-length survival motor neuron (SMN) protein.
Current treatments for SMA aim to increase SMN protein levels.

Purpose of the Study:

To evaluate the efficacy and safety of risdiplam, an oral SMN2 splicing modifier, for treating SMA.
To assess motor function improvements and long-term treatment effects of risdiplam in various SMA types.

Main Methods:

Phase 2/3 clinical trials were conducted.
Risdiplam was administered to infants and patients aged 2-25 years with SMA types 1, 2, or 3.
Motor function was assessed, and treatment effects were monitored for up to two years.

Main Results:

Risdiplam demonstrated significant improvements in motor function in infants with SMA type 1.
Patients aged 2-25 years with SMA types 2 or 3 also showed significant motor function improvements.
These motor improvements were sustained with up to two years of risdiplam treatment.
Risdiplam was generally well-tolerated, exhibiting a favorable benefit-to-risk profile.

Conclusions:

Risdiplam is an effective oral treatment for SMA across a broad age range and different SMA types.
Its ability to increase SMN protein production offers a valuable therapeutic option.
The oral administration of risdiplam enhances treatment convenience for patients with SMA.