IPS HEART has been awarded Orphan Drug Designation (ODD) by the FDA for GIVI-MPCs based on its unique ability to create new ...

Wave received supportive initial feedback from FDA, who recognize the severity of HD and are receptive to and engaged with Wave regarding a potential pathway to accelerated approval. FDA is open to ...

In both affected siblings, an accumulation of amorphous material immunoreactive for desmin, dystrophin and vimentin was observed in the cytoplasm of the skeletal muscle cells. An uncle and the ...

A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for ...

New study reveals crucial protein interactions in Duchenne Muscular Dystrophy, opening doors to more precise and effective ...

A new study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for understanding and ...

A new study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its ...

Entering 2025 having demonstrated rapid translation of genetic insights to positive clinical data; advancing clinically validated and multi-modal pipeline with the potential to treat well over 100 mil ...

Capricor's biologics license application for deramiocel (CAP-1002) is draws largely on the Phase 2 HOPE-2 trial and its ...

Here are the top medical news for the dayUnderstanding Duchenne Muscular Dystrophy: Study Unveils Critical Protein ...

Ananda Ghosh, an analyst from H.C. Wainwright, maintained the Buy rating on Avidity Biosciences (RNA – Research Report). The associated price ...

These include gene-based therapies (e.g., replacing a patient's faulty DMD genes with normally functioning ones), cell-based therapies (e.g., replacing dystrophin-deficient muscle cells with stem ...