Risdiplam is an oral bioavailable messenger ribonucleic acid (mRNA) splicing modifier used in the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older. Spinal muscular atrophy (SMA) is a severe and progressive congenital neuromuscular disease resulting from mutations in the survival of motor neuron 1 (SMN1) gene responsible for making SMN proteins. Clinical features of SMA include degeneration of motor neurons in the spinal cord which ultimately leads to muscular atrophy and, in some cases, loss of physical strength. Survival of motor neuron (SMN) proteins are expressed ubiquitously throughout the body and are thought to hold diverse intracellular roles in deoxyribonucleic acid (DNA) repair, cell signaling, endocytosis, and autophagy. A secondary SMN gene (SMN2) can also produce SMN proteins, but a small nucleotide substitution in its sequence results in the exclusion of exon 7 during splicing in approximately 85% of the transcripts which means that only approximately 15% of the SMN proteins produced by SMN2 are functional, which is insufficient to compensate for the deficits caused by SMN1 mutations. Emerging evidence suggests that many cells and tissues are selectively vulnerable to reduced SMN concentrations, making this protein a desirable target in the treatment of SMA.
Affygility Solutions has an occupational exposure limit (OEL) and control band assignment for this active pharmaceutical ingredient (API). This monograph also contains the acceptable daily exposure (ADE) value.
OEL Fastrac monographs are a cost-effective and convenient way to meet the requirements for PDEs (ADEs) contained in the EMA Guideline on setting health based exposure limits for use in risk identification in the manufacture of different medicinal products in shared facilities (EMA/CHMP/CVMP/SWP/169430/2012), as well as PIC/S and ANVISA requirements, and to obtain the following information:
To order an OEL/ADE monograph for Risdiplam, just click the ADD TO CART button.
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Affygility Solutions’ OEL Fastrac service is an user-friendly system that enables us to make confident decisions and focus on moving the compound safely through our facility. We can quickly access the database to determine if a new drug is within the OEL Fastrac library, and if so, immediately download a detailed OEL banding report. The OEL Fastrac system has been a helpful tool in our overall EH&S program.