Clinical Trials

Creatine kinase (CK), also known as creatine phosphokinase (CPK) or phosphocreatine kinase, is an enzyme expressed by various tissues and cell types. CK catalyses the conversion of creatine and uses adenosine triphosphate (ATP) to create phosphocreatine (PCr) and adenosine diphosphate (ADP). This CK enzyme reaction is reversible and thus ATP can be generated from PCr and ADP.
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Simply Stated: RNA-Based Therapies in Development for Myotonic Dystrophy

By Sujatha Gurunathan | January 30, 2025
Titled book Amyotrophic Lateral Sclerosis along with medical equipment, Title of book invented and fictitious made in Photoshop, conceptual image
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Simply Stated: Updates in Amyotrophic Lateral Sclerosis (ALS)

By Sujatha Gurunathan | October 29, 2024
Genetic engineeering concept with 3d rendering dna helix and a part of dna
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Next-Generation Exon-Skipping Therapies May Change the DMD Landscape

By Myrna Traylor | September 13, 2024
A father, mother, and three sons sit on an outdoor patio step
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My Gene Therapy Journey: The Curran Family

By Rebecca Hume | September 5, 2024
Parents hold their four-year-old daughter, who received Zolgensma for spinal muscular atrophy as an infant.
Blog Post

A Revolutionary Decade for Spinal Muscular Atrophy Therapies

By Chris Anselmo | August 1, 2024
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Clinical Trial Alert: Phase 2 Pivotal Study of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy

By Sujatha Gurunathan | June 17, 2024
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MDA Ambassador Guest Blog: Finding Hope and Optimism in New Treatments

By Chase Rankin | June 4, 2024
Woman in electric wheelchair waits for consultation with doctor in clinic waiting area. Nurse invites patient with disability on procedures. Medical staff and people in modern medical facility lobby.
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Treatment Options for Autoimmune Neuromuscular Diseases

By Patrick Sullivan | May 17, 2024
Illustration of digital health technologies
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Can Wearables and Smartphones Make Clinical Trials Better?

By Andrew Zaleski | May 9, 2024
Ribonucleic acid strands consisting of nucleotides important for protein bio-synthesis - 3d illustration
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Simply Stated: Updates in SELENON-Related Myopathy

By Sujatha Gurunathan | May 7, 2024