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Scholar Rock Provide Update on Phase 2 TOPAZ trial of Apitegromab

Treatments & Research
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We are excited to share with you our brand refresh and new website!

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Ian, Mark, Neil & Paul’s LEJOG Challenge

Fundraising

Taking the leap for SMA UK

Fundraising

Say hello to Gary!

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APPG for Muscular Dystrophy: Newborn Screening for Rare Conditions

Advocacy
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SMA UK Trustee Event – An evening with Sir Richard Dearlove OBE

Fundraising
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Calling for Progress on Care Coordination in the UK

Advocacy
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Novartis presents data on long-term treatment with Zolgensma

Information
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Treatments & Research

Taldefgrobep Alfa Receives FDA Fast Track Designation for SMA

Information
Treatments & Research