Clinical Trials

For SARCOGLICANOPATHIES


LGMD2E OR LGMDR4

In 2018  the first clinical trial of gene therapy for LGMD2E or LGMDR4 started in the United States.

Gene Delivery Clinical Trial of SRP-9003 for Patients With LGMD2E (Beta-sarcoglycan Deficiency) 

 

LGMD2D OR LGMDR3

On 2013 a gene therapy clinical trial for LGMD2D or LGMDR3 started in the United States carried out by dr. Jerry Mendell.

Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA

 

LGMD2C OR LGMDR5

In 2011, a gene therapy clinical trial was carried out in France for LGMD2C or LGMDR5 by dr. Serge Herson.

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C 

 

LGMD2D OR LGMDR3

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)

 

PRESS RELEASES 2020 - CLINICAL TRIALS

| Clinical trials

15 October 2020 | Clinical trials

SRP-9003 Gene Therapy for LGMD2E Shows Benefit after 1 year - Muscular Dystrophy News Today - June 9 2020

https://musculardystrophynews.com/2020/06/09/srp-9003-gene-therapy-continues-significant-benefits-children-lgmd2e-phase-1-2-trial/?fbclid=IwAR14O20MUNtLs2wuIwz2oH4CVZivqhmP4IC3ExbKXsZmJXr1H3n6C9Fd4MY 

New data gives hope for Limb Girdle Muscular Dystrophy gene therapy - Patient Worthy - June 17, 2020

https://patientworthy.com/2020/06/17/data-gives-hope-limb-girdle-muscular-dystrophy/

PRESS RELEASES 2019 - CLINICAL TRIALS

| Clinical trials

31 December 2019 | Clinical trials

SRP-9003 gene therapy shows "very encouraging" results at 9 months in girdle dystrophy - Sarepta Reports - october 9 2019

https://musculardystrophynews.com/2019/10/09/srp-9003-lgmd-2e-gene-therapy-shows-encouraging-9-month-results-sarepta-reports/?fbclid=IwAR2eeni-mgYqgcTPkfFT8aTXsRg6I0WAymm6OENZvBM098ozSKo70btfZ3Q

PRESS RELEASES 2018 - CLINICAL TRIALS

| Clinical trials

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