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Rare Disease Day

Rare Disease Day

At the kind invitation of Dr. Alberto Malerba, Lecturer in Gene Therapy at the Royal Holloway, University of London, Rajeshri Badiani presented the FSHD UK journey Rajeshri Badiani presented the FSHD UK journey on ‘starting a volunteer group to setting up a formal...

Experimental Therapy SAT-3247 Shows Promise in FSHD Mouse Study

Experimental Therapy SAT-3247 Shows Promise in FSHD Mouse Study

A new experimental therapy designed to support muscle regeneration has shown encouraging results in a mouse model of facioscapulohumeral muscular dystrophy (FSHD), offering fresh hope for future treatment approaches for the condition. The therapy, known as SAT-3247,...

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