There are various scientific research approaches to finding a cure for Limb girdle muscular dystrophies. It is also important to develop adequate natural history studies in parallel. Clinical trials and natural history studies are vital in advancing research to help find a treatment and cure for LGMD.
This is a searchable registry and database of federally supported clinical trial results and private conducted in the United States and around the world.
Here you can view all scientific publications on Limb girdle muscular dystrophies and other diseases.
There are three gene therapy studies for sarcoglycanopathies.
- A study has been ongoing since 2011 on LGMD2E in the United States, there are two publications on the preclinical phase of prof. Jerry Mendell and Louise Rodino Klapac.
The first clinical trial on LGMD2E is underway.
The gene therapy study for LGMD2D was developed in the United States and there are 4
scientific publications (2008-2009-2010-2020) by dr. Jerry Mendell.
Another study is underway on LGMD2C in France, there are two scientific publications by
prof. Isabelle Richard. A clinical trial took place in 2011.
A study was published in 2012 by prof. Giulio Cossu.
A study was published in the United States by Prof. Elizabeth M McNally.
In 2018, the first clinical trial of gene therapy for LGMD2E started in the United States.
Gene Delivery Clinical Trial of SRP-9003 for Patients With LGMD2E (Beta-sarcoglycan Deficiency)
On 2013 a gene therapy clinical trial for LGMD2D started in the United States carried out by dr. Jerry Mendell.
Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA
In 2011, a gene therapy clinical trial was carried out in France for LGMD2C
Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
On 2007, a gene therapy clinical trial was carried out in the United States by Dr. Jerry Mendell
Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)