Explore a decade of impactful milestones in the fight against Limb-Girdle Muscular Dystrophy (LGMD). From groundbreaking genetic sequencing and awareness campaigns to international conferences and clinical trials, discover the significant advancements and ongoing efforts to improve the lives of those affected by LGMD.

2014

Free next-generation genetic sequencing for LGMDs (35 genes) made available through an LGMD advocacy program to US patients resulting in thousands of free tests and genetic diagnosis

2014 Website

LGMD-Info.org, the “Information Hub for the LGMD community” was created

2014 Limegreen

Lime Green for LGMD awareness campaign introduced

Biomarker clinical research study of individuals with dysferlinopathy

2015

2015awarenessday

First LGMD Awareness Day was celebrated

First State Proclamations received declaring September 30th Limb-Girdle Muscular Dystrophy Awareness Day in Wisconsin, Washington, Missouri, and Colorado

Dysferlin Scientific Conference, Toronto, Canada

2B Empowered Patient Conference, Dallas, Texas

2016

2016 Enmc

219th ENMC International Workshop Titinopathies International database of titin mutations and phenotypes, Heemskerk, The Netherlands

The Samantha J. Brazzo Foundation changes to CureLGMD2i

CureLGMD2i and LGMD2i Research Fund support the first-ever clinical trial in LGMD2I/R9 A Trial of PF-06252616 in Ambulatory Participants With LGMD2I, led by Dr. Kathryn Wagner

Second 2B Empowered Patient Conference, Dallas, Texas

Dysferlin Registry launched a new platform for genetically confirmed dysferlinopathy patients

Dual vector gene therapy for dysferlinopathy reaches Investigational New Drug (IND) FDA approval

2017

2017myonexuslogo

Myonexus Therapeutics was founded to develop gene therapies to treat five subtypes of LGMD (2B/R2, 2C/R5, 2D/R5, 2E/R4, and 2L/R12)

Revised nomenclature (classification) system for LGMDs created

First Intra-muscular (IM) dual vector gene therapy clinical trial for dysferlinopathy

2018

2018grasp

The Genetic Resolution and Assessments Solving Phenotypes in LGMD (GRASP-LGMD) consortium was created to hasten therapeutic development in the LGMDs.

LGMD2L Foundation was established

Coalition to Cure Calpain 3 announced their Gene Therapy Initiative to accelerate the development of gene therapies to treat LGMD2A/R1

2018lgmd2a R1

LGMD2A/R1 Scientific Conference in Arlington, Virginia

LGMD-1D DNAJB6 Foundation was established

First whole-body administration of gene therapy for LGMD (beta-Sarcoglycan – LGMD2E) (Sarepta Therapeutics)

2019

International LGMD Community Advisory Board (CAB) established

2019limeemoji Web

“LIMEmoji Challenge” to help raise awareness of LGMD was introduced

Clinical Study for Open Label Safety and Efficacy of Once Weekly Steroid in Patients with LGMD

2019conference

First International LGMD Conference in Chicago, Illinois

2109india

2B Empowered patient meeting in Mumbai, India

2020

2020laf

LGMD Awareness Foundation was established

2020girdie

Girdie, the LGMD Ambassador, was introduced to the LGMD community.

Patient Listening Session for LGMD with the FDA

2021

2021lgmdnews

First issue of LGMD News magazine was published (vol 1/issue 1)

First “Virtual” International LGMD Conference

2021diagnosticcodes

Dedicated ICD-10 medical codes approved for some of the LGMD subtypes and for non-specified subtypes

Dosing of First Patient in Phase 2 Trial of BBP-418 (Ribitol) in Limb- Girdle Muscular Dystrophy Type 2i (LGMD2I/R9) (BridgeBio Pharma and Affiliate ML Bio Solutions)

2022

2022summit Web

LGMD Global Advocacy Summit convened.

2022girdie

Girdie, the LGMD ambassador, was “brought to life” and debuted at the Wellstone Dystroglycanopathies Patient & Family Conference in Iowa.

2022externallyled

First Externally Led Patient-Focused Drug Development Meeting (EL-PFDD) for LGMDs with the FDA and other stakeholders

CDC institutes LGMD-specific ICD-10 Codes

The nonprofit Team Titin, Inc. was formed, which supports the LGMD2J/R10 community.

Daniel Ferguson LGMD Foundation was established in Australia

Team Titin hosted the SciFam Research-Family Conference.

First Patient Dosed in Phase 1/2 Clinical Trial in Europe of Gene Therapy for LGMD-R9 (Atamyo Therapeutics)

CureLGMD2i hosts the first “Connecting for a Cure” event on LGMD Awareness Day

The Clinical Outcome Study/Natural History of Dysferlinoapthy celebrates 10 years of patient participation and clinical data collection

LGMD Advocacy Bundles deployed at 6 clinics in the US by LGMD Awareness Foundation

2023

2023voicepatient

LGMD “Voice of the Patient” Report was published highlighting the unmet needs of six LGMD subtypes

Study initiated to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients with Limb-Girdle Muscular Dystrophy 2I (LGMD2I/R9) (ML BioSolutions)

First Patient treated in Phase 1 / Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I/R9 (AskBio)

2023intlconference

First “hybrid” International LGMD Conference in Washington, DC

Coalition to Cure Calpain 3 launches new LGMD2A/Calpainopathy Registry

The nonprofit The Dion Foundation was established

First whole body/systemic treatment of SRP 6004 Proof of Concept Clinical Trial, NAVIGENE study of dysferlinopathy (Sarepta Therapeutics)

2024

Screening initiated in EMERGENE, a Phase 3 Clinical Study of SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E/R4 (Sarepta Therapeutics)

2024drugworkshop

LGMD Scientific Drug Development Workshop in Bethesda, Maryland

Clinical trial application approved in France and Italy for testing ATA-200, an investigational gene therapy for children with limb-girdle muscular dystrophy type 2C/R5 (Atamyo Therapeutics)

2024 European

First LGMDR9 (LGMD2I) European Patient Day in Amsterdam, The Netherlands

LGMD2A/R1 Scientific Meeting in Arlington, Virginia

Dysferlin Scientific Conference, Houston, Texas

2024

10th Annual LGMD Awareness Day is celebrated worldwide