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THE GENE THERAPY PROJECT FUNDED BY  GFB ONLUS

 

 


 

 

POSITIVE DATA FOR SRP-9003

 

June 8th  2020: Sarepta Therapeutics Announces Positive Expression and Functional Data From the SRP-9003 Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type 2E.

 

 

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Other press releases:

New data gives hope for Limb Girdle Muscular Dystrophy gene therapy - Patient Worthy

SRP-9003 gene therapy for LGMD2E shows benefits after 1 year - Muscular  dystrophy news today

 

 


 

 

 

 

COVID-19 RESOURCES FOR MUSCULAR DYSTROPHY COMMUNITY

 

FROM THE SPEAK FOUNDATION:

 

In the website of the Speak Foundation www.thespeakfoundation.com you can find:

- COVID-19 Advocacy Letter for Individuals with MD

- Advocacy Letter to Healthcare Providers

- COVID-19 Preparedness Checklist       It is extremely important that you gather the following information and have it readily available if you should become ill from COVID-19. You should also make sure your family members and all caregivers know where this information is kept in the event you require going to the hospital for health issues related to COVID-19. Keep it in a safe and easily accessible location.

 

 

FROM EURORDIS

 

In the website of Eurordis  https://www.eurordis.org/content/eurordis-press-releases you can find:

- EURORDISstatement_COVID19Triage

EURORDIS-Rare Diseases Europe would like to bring to their urgent attention the concerns and needs of people living with a rare disease, their families and carers during the COVID-19 crisis. Through this open letter, we implore policy makers and authorities in Europe and around the world to take action to protect people living with a rare disease from becoming even more vulnerable during this crisis in line with the recommendations set out.

 

 


 

 

 

CORONAVIRUS: informations for MD patients

 

Here you can find PPMD COVID-19 (CORONAVIRUS) INFORMATION CENTER

 

 



 

ALL THREE PATIENTS WAS DOSED IN HIGH-DOSE COHORT FOR LGMD2E

 

SAREPTA ANNOUNCED:  "we have dosed all three patients now in our high-dose cohort for LGMD2E. We will have expression and safety results available in the second quarter, and we anticipate announcing that data at an appropriate medical meeting in the second quarter".

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Gene Therapy SRP-9003 Showing Very Encouraging

 

Results at 9 Months in LGMD , Sarepta Reports

 

New nine-month data from a Phase 1/2 trial show that a single administration of the gene therapy SRP-9003 (formerly, MYO-101) at low dose significantly improved functional measures and lowered the levels of a biomarker of muscle damage in three children with limb girdle muscular dystrophy (LGMD) type 2E.

The trial (NCT03652259) into the therapy, being developed by Sarepta Therapeutics (after it acquired Myonexus Therapeutics), is still recruiting patients at its single site in Ohio.

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Here are the links to the videos from today's Sarepta LGMD2E Functional Data Call. Impressive to say the least...

100M Running

Trunk Control

Getting Up From Setting

 


 

 

 

CLINICAL UPDATE : MYO-101 FOR LGMD TYPE 2E

 

Business Update : Myonexus acquisition

February 27, 2019

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OTHER PATIENTS AFFECTED FROM LGMD2C-2D-2E  IN GFB ONLUS

 

In the last months GFB ONLUS has found other patients affected from Lgmd2c-2d-2e. GFB counts now a total of 403 patients affected from Sarcoglycanopathy, so divided:


LGMD2C

LGMD2D

LGMD2E

LGMD2F

SARCOGL.

2010

0

1

5

0

 

2013

4

14

14

1

 

2014

9

27

21

1

 

2015

12

50

25

1

 

2016

23

77

69

1

3

2017

37

104

97

4

3

2018 132 152 112 4 3

 

On the website of the association you can find the list of the patients sorted by geographical provenance to the link :

http://www.beta-sarcoglicanopatie.it/index.php?option=com_content&view=article&id=46&Itemid=54

 


 

 

 

SUPPORTERS

 

2020

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OTHER SUPPORTERS  2018-2017-2016-2015-2014-2013

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