FDA Releases Guidance on Treatment for Duchenne Muscular Dystrophy
The agency issues draft guidance on the development of drugs to treat Duchenne muscular dystrophy.
FDA released draft guidance on June 9, 2015 that will assist drug companies in the clinical development of drugs to treat X-linked Duchenne muscular dystrophy (DMD) as well as Becker muscular dystrophy, DMD-associated dilated cardiomyopathy, and symptomatic carrier states in females. The guidance does not address treatments for secondary complications of muscle degeneration in dystrophinopathies.
In developing the draft guidance, FDA took into consideration a proposed draft guidance written by the Parent Project Muscular Dystrophy (PPMD) advocacy group as well as public comments to the PPMD guidance. FDA states that the collaboration with PPMD is an example of how input from stakeholders, patients, and caregivers can be beneficial to drug development. “FDA values PPMD’s effort and input and appreciates the insights provided by the DMD community.”
Source: FDA.gov
Drug Shortages and Complying with FDA’s 21 CFR 211.110 Guidance
April 2nd 2025Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates, and Rona LeBlanc-Rivera, PhD, principal consultant, Regulatory Affairs at Regulatory Compliance Associates, answer some questions about FDA’s January 2025 21 CFR 211.110 guidance document.
AES Clean Technology Launches Next-Generation OSM Utility Solution for Cleanrooms at INTERPHEX 2025
April 2nd 2025Officially launched at INTERPHEX 2025, the Omni ASCENT is a next-generation off-site manufactured vertical utility solution that offers optimized cleanroom flexibility and efficiency.
