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2 clusters · 5 sources · 14 days · First seen · Last updated

Duchenne muscular dystrophy treatment developments

Overview

The landscape for Duchenne muscular dystrophy (DMD) treatments is experiencing both clinical advancements and significant administrative hurdles.

On the clinical front, therapeutic approaches are diversifying. Capricor Therapeutics is amending its FDA filing for the cell therapy deramiocel, shifting its focus toward upper limb skeletal muscle function following Phase 3 HOPE-3 trial data. Simultaneously, the HDAC inhibitor givinostat is moving toward conditional marketing authorization in the European Union, targeting the inflammatory and fibrotic processes of the disease rather than dystrophin replacement.

However, patient advocacy groups are highlighting systemic barriers to accessing these and other innovations. In Spain, the Duchenne Parent Project España (DPPE) has reported severe delays, noting that patients face an average wait of over 500 days for essential therapies, with some orphan drugs taking more than 700 days to become available. Advocates are calling for more equitable access to multidisciplinary care and the elimination of geographic inequalities.

Entities

Duchenne Parent Project España · Silvia Ávila · Food and Drug Administration · Fuengirola · Capricor Therapeutics

Timeline

  1. 9 days ago

    [HEALTH] 2 sources
    Duchenne muscular dystrophy advocates highlight treatment delays in Spain

    On World Duchenne Awareness Day, advocates in Spain highlight critical delays in treatment access, with patients waiting over 500 days for innovative therapies for the rare muscular dystrophy.

  2. 22 days ago

    [HEALTH] 3 sources
    Duchenne muscular dystrophy treatments see regulatory and clinical shifts

    New developments in Duchenne muscular dystrophy treatments include Capricor Therapeutics pivoting its FDA filing for deramiocel toward upper limb function and the EU moving toward authorization for givinostat.

Sources

consalud.es · gazeta.eu · lanocion.es · newsable.asianetnews.com · world-today-news.com