Help First ION283 Safety Study, Save 10 Children

Please Give to support the first-ever Safety Study for Lafora disease. Change the lives of 10 patients. Change the future for Gajic, Malaj, Durmisevic, Salkic, Mrvosevic and many more families around the world affected by Lafora Disease.

Be part of the fight against one of the world's most severe childhood diseases. Save children's lives!

After 30 Years of frustration, we face a pivotal opportunity. Under Professor Dr. Berge Minassian’s leadership, a team at UT Southwestern Dallas,TX will start a Safety Study of ION283 approved by FDA , a potential treatment, later this year. The first-ever study is only possible because of your generosity, though. We need to raise $1.5 million to get it off the ground. Your gift will bring hope to life for 10 patients and their loved ones. Please give if you can and spread the word—time is of the essence, and everyone can make a difference. Can you keep hope alive?  This is an exciting step forward! It will ensure a brighter future for the entire Lafora community.

Lafora disease is a severe form of progressive myoclonic epilepsy manifested by generalized seizures, myoclonus, intellectual decline, ataxia, spasticity, dysarthria, visual loss, and, in later stages, psychosis and dementia.Most patients are asymptomatic until adolescence, but they undergo first insidious, then rapid progressive myoclonus epilepsy toward a vegetative state and death within a decade.Patients usually present between the ages of 11 and 18 years. Progression of the disease varies, but total disability or death usually occurs within 10 years of symptom onset due to complications of central nervous system (CNS) degeneration and status epilepticus. Generally, the course of Lafora disease is progressive and characterized by increasing frequency and intractability of seizures. 

Please share and support Lafora families and Give Hope for 10 Children !

Thank You ALL from the bottom of our hearts for building a brighter future for the Lafora community!

With Gratitude,

Marina Weiss & Lafora Families ❤️

Molimo vas da date podršku prvoj studiji bezbednosti za Lafora bolest. Promenite živote 10 pacijenata. Promijenite budućnost za Gajić, Malaj, Durmišević, Salkić, Mrvošević i mnoge druge porodice pogođene Laforinom bolešću. 

 

Budite deo borbe protiv jedne od najtežih dečjih bolesti na svetu. Spasite dečije živote! 

 Posle 30 godina frustracije, suočavamo se sa ključnom šansom. Pod vođstvom Profesora Dr Berge Minassiana, tim u UT Southvestern Dallas, Teksas - će kasnije ove godine započeti Studiju bezbednosti ION283 koju je odobrila FDA, potencijalni tretman. Međutim, prva studija je moguća samo zbog vaše velikodušnosti. Moramo da prikupimo 1,5 miliona dolara da bismo ga pokrenuli. Vaš poklon će oživeti nadu za 10 pacijenata i njihovih najmilijih. Molim vas dajte ako možete i proširite reč – vreme je od suštinske važnosti i svako može da napravi razliku. Možete li održati nadu živom? Ovo je uzbudljiv korak napred! To će obezbediti svetliju budućnost za celu Lafora zajednicu. 

 Lafora bolest je teški oblik progresivne mioklonične epilepsije koja se manifestuje generalizovanim napadima, mioklonusom, intelektualnim opadanjem, ataksijom, spastičnošću, dizartrijom, gubitkom vida i, u kasnijim fazama, psihozom i demencijom. Većina pacijenata je asimptomatska do prve adolescencije, ali podleže podmukla, zatim brzo progresivna mioklonusna epilepsija ka vegetativnom stanju i smrt u roku od jedne decenije. Pacijenti se obično javljaju u dobi između 11 i 18 godina. Progresija bolesti varira, ali potpuni invaliditet ili smrt se obično javljaju u roku od 10 godina od pojave simptoma zbog komplikacija degeneracije centralnog nervnog sistema (CNS) i epileptičnog statusa. Generalno, tok Laforine bolesti je progresivan i karakteriše ga sve veća učestalost i nepopravljivost napada.

 Molimo Vas da podelite i podržite porodice Lafora i dajte nadu za 10 dece! 

Hvala Vam SVIMA unapred od srca za izgradnju svetlije budućnosti za zajednicu Lafora!!!!!

Sa Puno poštovanja i mnogo zahvalni,

Marina Weiss & Lafora Porodice❤️