Scientific Research

For Sarcoglycanopathies


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.

 

ClinicalTrials.gov

This is a searchable registry and database of federally supported clinical trial results and private conducted in the United States and around the world.

https://clinicaltrials.gov/

Pubmed

Here you can view all scientific publications on Limb girdle muscular  dystrophies and other diseases.
https://pubmed.ncbi.nlm.nih.gov/

 


GENE THERAPY

There are three gene therapy studies for sarcoglycanopathies.

LGMD2E
- 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.

gene therapy

The american project about LGMD2E


The first clinical trial on LGMD2E is underway.
Read More

LGMD2D
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.
Read more 

LGMD2C
Another study is underway on LGMD2C in France, there are two scientific publications by
prof. Isabelle Richard. A clinical trial took place in 2011.
Read More

 

Cell Therapy

A study was published in 2012 by prof. Giulio Cossu.

Cell Therapy

 

Exon Skipping Therapy

A study was published in the United States by Prof. Elizabeth M McNally.

Exon Skipping Therapy

 

CRISPRCAS9 ThERAPy 

There is an ongoing study for LGMD2i and LGMD2G in the United States.

Read More

CrisprCas9 Therapy

 

 

Clinical Studies

LGMD2E

There are two natural history studies for LGMD2E, one in the United States and one in Milan in Italy.

United States

Milan

LGMD2C-2D-2E
There is a natural history study for LGMD2C-2D-2E in the United States

United States

 

 

Clinical Trials

LGMD2E

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)

Read more

LGMD2D

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
Read more

LGMD2C

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 

Read more

LGMD2D

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)
Read more

 

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