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THE GENE THERAPY PROJECT FUNDED BY  GFB ONLUS

 

 


 

 

"MUSCLE DIVERSITY": THE CARLES SANCHEZ RIERA PROJECT

 

Carles Sanchez Riera is starting an interesting project on LGMD Limb Girdle Muscular Dystrophies, entitled "Muscular Diversity",  at the "La Sapienza University" of Rome, in Italy.

Carles has a limb girdle muscular dystrophy and his aim is to understand the differences among muscles of the same patient: "Nothing is black or white, there are many muscles of our body that work appropriately, they could have the key cues to find a treatment."

https://sites.google.com/view/muscle-diversity-project/

 

 

 


 

 

 

 

The GFB on National Television for the seventh time

 

On July 24, Rai Parlamento broadcast the seventh interview of the GFB on the Italian national television, entitled "Beta-sarcoglicanopatie: research and telemedicine".

watch the video


 

 

 


 

 

 

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.

 

 

Read more

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

 

 


 

 

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
2020 158 206 141 5 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

1.000 €

Donazione NN per la ricerca scientifica

15.104,23 Contributo 5x1000 relativo al 2018
50 €

Donazione NN per la ricerca scientifica da Salerno

120 €

Donazione NN per la ricerca scientifica

15 € Donazione NN per la ricerca scientifica
20 € Donazione NN per la ricerca scientifica
10 € Donazione NN per la ricerca scientifica dalla Spagna
2000 € Donazione NN per la ricerca scientifica da Lecco
150 € Donazione NN per la ricerca scientifica
30 € Donazione NN per la ricerca scientifica
200 € Donazione NN per la ricerca scientifica
100 € Donazione NN per la ricerca scientifica

 

 

2019

300 € Donazione NN per la ricerca scientifica
1.000 € Donazione NN per la ricerca scientifica da Roma
150 € Donazione NN per la ricerca scientifica
200 € Donazione NN per la ricerca scientifica
13.424,23 Contributo 5x1000 relativo al 2017
150 € Donazione NN per la ricerca scientifica
20 € Donazione NN per la ricerca scientifica
50 € Donazione NN per la ricerca scientifica da Treviso
85,21 € Donazione Pancaffè per la ricerca scientifica
250 € Donazione coscritti 1949 di Talamona in memoria di Vola Bruno
2.945 € Donazione Memorial Simone Alberton da Treviso
300 € Donazione NN per la ricerca scientifica da Roma
200 € Donazione NN per la ricerca scientifica
1.130 € Donazione NN per la ricerca scientifica da Como
135 € Donazione coscritti 1939 di Talamona in memoria di Tirinzoni Guido
600 € Donazione NN per la ricerca scientifica
300 € Donazione NN per la ricerca scientifica da Viareggio
50 € Donazione NN per la ricerca scientifica da Roma
300 € Donazione NN per la ricerca scientifica da Roma
1.000 € Donazione NN per la ricerca scientifica
2.000 € Donazione NN per la ricerca scientifica da Lecco
110 € Donazione coscritti 1964 di Talamona
150 € Donazione NN per la ricerca scientifica
300 € Donazione NN per la ricerca scientifica da Lecco
250 € Donazione NN per la ricerca scientifica
150 € Donazione NN per la ricerca scientifica da Roma
25.549,44 TOTALE 2019

 

 

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

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