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Deficit di ADSL, Cerchiamo La Cura!

€3,661 of €50,000 goal

Raised by 37 people in 5 months

Siamo tutti bambini con una malattia rara metabolica che si chiama "carenza dell'enzima Adenilsuccinato liasi"
Nelle forme più gravi non riusciamo a superare i primi anni di vita, negli altri casi soffriamo tutti di epilessia farmaco resistente, quasi nessuno di noi è in grado di camminare autonomamente e non riusciamo a parlare.
Ad oggi per noi non esiste cura .
Viviamo in diverse parti del mondo, ma i nostri genitori si sono messi in contatto e uniti per sostenere i progetti di ricerca che ritengono piu' validi su questa malattia rara per tentare di trovare una terapia.
Il progetto di ricerca che attualmente finanziamo con questo gofundme è quello diretto dal Professor Francesco Cecconi ,responsabile del centro "Unit of cell stress and survival, Danish Cancer Society Research center of Copenhagen"; il Francesco Cecconi è anche Professore del dipartimento di Biologia dell'università di Roma Tor Vergata in Italia.
Il progetto di ricerca si suddivide in più fasi e per portare avanti la ricerca  sono necessari circa 50'000 Euro l'anno, comprensivi di: spese per l'acquisto  dei reagenti, del rimborso spese per i ricercatori, costi per le prime fasi di sperimentazione sulle cellule e  clinica.
L'intero progetto di ricerca prevede studi che dureranno per un periodo di 4 anni.
Per l'anno 2018 abbiamo raccogliere EUR 50.000!

La ricerca per noi è l'unica possibilità per poter sperare di avere una cura e migliorare così la nostra vita.
Grazie del vostro aiuto da tutti noi bambini provenienti da tutte le parti del mondo: Marta (Poland), Avery (USA), Matthew (Ireland), Jude (Israel), Gia (USA), Szymon (Poland), Carola (Italia), David (Poland), Jack (USA), Sydney e Carson (USA), Hamish e William (Australia), Cyrus (USA), Taft (USA) Gabriela (Brazil)

We are children with a rare metabolic disorder called "Adenylosuccinate Lyase Deficiency". In the most severe forms, we can't survive the first years of life. In other cases, we fight against drug resistant epilepsy, while we struggle to learn to walk and talk. There is currently no viable cure or treatment for our condition.

We live in different parts of the world, but our parents have come together to support the research projects that show the most promise in understanding this disorder and developing an effective therapy.

All money raised from this fundraiser will be donate to Professor Francesco Cecconi, head of the Center for Cell Stress and Survival and of the Danish Cancer Society Research Center of Copenhagen. Francesco Cecconi is also a Professor of the Department of Biology at the University of Rome Tor Vergata in Italy.

The research project is divided into several phases, and to continue the research we will need approximately 50,000 Euros a year, including: reagent purchase costs, reimbursement of costs for researchers, costs for early stages of research on cells and clinical trials. The entire research project includes studies that will last for 4 years.

The first year of the study is already underway, but for the year 2018 we need to collect 50,000 Euros and the help of all our friends, acquaintances, and new supporters is crucial to us!

These research studies are the only chance we can hope to develop a treatment and thus improve our the quality of our lives. These children from all over the world thank you for all your support: Marta (Poland), Avery (USA), Matthew (Ireland), Jude (Israel), Gia (USA), Szymon (Poland) (Brazil), Jack (USA), Sydney and Carson (USA), Hamish and William (Australia), Cyrus (USA), Taft (USA), Gabriela (Brazil).
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An article of the online review “FarmaMagazine” about Carola and Adsl deficiency
+ Read More
We are children with a rare metabolic disorder called "Adenylosuccinate Lyase Deficiency". In the most severe forms, we can't survive the first years of life. In other cases, we fight against drug resistant epilepsy, while we struggle to learn to walk and talk. There is currently no viable cure or treatment for our condition.

We live in different parts of the world, but our parents have come together to support the research projects that show the most promise in understanding this disorder and developing an effective therapy.

All money raised from this fundraiser will be donate to Professor Francesco Cecconi, head of the Center for Cell Stress and Survival and of the Danish Cancer Society Research Center of Copenhagen. Francesco Cecconi is also a Professor of the Department of Biology at the University of Rome Tor Vergata in Italy.

The research project is divided into several phases, and to continue the research we will need approximately 50,000 Euros a year, including: reagent purchase costs, reimbursement of costs for researchers, costs for early stages of research on cells and clinical trials. The entire research project includes studies that will last for 4 years.

The first year of the study is already underway, but for the year 2018 we need to collect 50,000 Euros and the help of all our friends, acquaintances, and new supporters is crucial to us!

You can make a donation using GOFUNDME

Or by donating directly to Piccoli Geni Onlus by bank transfer
On cc headed to: "Piccoli Geni Onlus"
Iban IT03Q0832703221000000004199
SWIFT / BIC: ROMAITRRXXX
Agency 21 Rome Piazza Nicosia
CAUSE: Adsl deficiency research

These research studies are the only chance we can hope to develop a treatment and thus improve our the quality of our lives. These children from all over the world thank you for all your support: Marta (Poland), Avery (USA), Matthew (Ireland), Jude (Israel), Gia (USA), Szymon (Poland) (Brazil), Jack (USA), Sydney and Carson (USA), Hamish and William (Australia), Cyrus (USA), Taft (USA), Gabriela (Brazil).
+ Read More
E' una grande impresa sostenere questo progetto di ricerca! i fondi che dobbiamo raccogliere entro l'anno 2018 non sono pochi ma siamo fiduciosi di poterci riuscire con il vostro aiuto, piccole e grandi donazioni, tante condivisioni chiedendo ai vostri amici di collaborare,in soli 4 giorni abbiamo ricevuto molte donazioni, continuiamo a fare tutto cio' che è nelle nostre possibilità per dare una speranza di cura a tutti i bambini con la malattia rara carenza di Adsl.
grazie Benedetta
+ Read More
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€3,661 of €50,000 goal

Raised by 37 people in 5 months
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€10
Łukasz Sujka
13 days ago
EG
€100
Enrico Guida
1 month ago
LS
€100
Lavi e Marco Sonnino
1 month ago
ŁS
€5
Łukasz Sujka
1 month ago
€10
Łukasz Sujka
1 month ago
€100
Anonymous
2 months ago
€125
Kari Payne Brown
2 months ago
VB
€50
Valentina Bavaresco
2 months ago
€10
Łukasz Sujka
2 months ago
FR
€200
Francesca Roma
2 months ago
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