“CureSCG's mission is to find a cure for LGMD Sarcoglycanopathy, inclusive of its LGMD2C, LGMD2D, LGMD2E, and LGMD2F variants.”
Learn More
Welcome to "Cure Sarcoglycanopathy," a platform born from a shared vision to impact the lives of those
with Limb Girdle Muscle Dystrophy (LGMD), specifically focusing on the Sarcoglycanopathy gene. Our
journey began with the need for a platform showcasing advanced research and acting as a catalyst for
vital funding dedicated to LGMD research. Founded with passion, "Cure SCG" is dedicated to unraveling
the mysteries of LGMD, aiming to find a cure. We emphasize collaboration, bringing together researchers,
clinicians, advocates, and the community to drive innovation and accelerate discovery.
At "Cure SCG," our mission is to foster collaboration, share ideas, and advance LGMD research, with a
focus on the Sarcoglycanopathy gene. We aim to propel the field forward and make significant strides
towards a cure through dynamic and inclusive knowledge-sharing.
At “Cure SCG”, our mission is clear: to provide a dynamic and inclusive space for the exchange of ideas, research findings, and advancements in the field of LGMD, particularly focusing on the Sarcoglycanopathy gene. By facilitating collaboration and knowledge-sharing, we strive to propel LGMD research forward and make significant strides towards a cure.
Limb Girdle Muscle Dystrophy poses unique challenges, and the urgency to find effective treatments has never been more critical. Our commitment to the cause stems from a deep understanding of the impact LGMD has on individuals and their families. By concentrating our efforts on the Sarcoglycanopathy gene, we aim to address a specific aspect of LGMD, paving the way for targeted therapies and, ultimately, a cure.
At the heart of Cure SCG is a commitment to empower researchers and inspire hope within the LGMD community. By providing a platform for the latest breakthroughs, clinical trials, and emerging therapies, we aim to catalyze progress and encourage a sense of optimism among those affected by LGMD.
ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5
Status : NOT YET RECRUITING
Sponsor: Atamyo Therapeutics
A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene
Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)
Status : COMPLETED
Sponsor: Sarepta Therapeutics, Inc.
Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
Status : COMPLETED
Sponsor: Nationwide Children's Hospital
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
Status : ACTIVE, NOT RECRUITING
Sponsor: Sarepta Therapeutics, Inc.
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
Status : ACTIVE, NOT RECRUITING
Sponsor: Sarepta Therapeutics, Inc.
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)
Status : RECRUITING
Sponsor: Sarepta Therapeutics, Inc.
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice Status : RECRUITING Sponsor: Sarepta Therapeutics, Inc.
Long-Term Development of Muscular Dystrophy Outcome Assessments (GRASP-01-005) Status : ACTIVE, NOT RECRUITING Sponsor: Virginia Commonwealth University
Natural History Study in Patients With LGMDR5/2c
Status : NOT YET RECRUITING
Sponsor: Atamyo Therapeutics
Clinical Determinants of Disease Progression in Patients With Limb Girdle Muscular Distrophy Type 2E (NeuroLGMD2E)
Status : COMPLETED
Sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinicos
Limb Girdle Muscular Dystrophy Type 2E Recruitment Study
Status : RECRUITING
Sponsor: Nationwide Children's Hospital
Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E
Status : RECRUITING
Sponsor: Linda Pax Lowes
