First Drug Targeting Core Genetic Defect in Friedreich's Ataxia
affects one in 50,000 people, often shortening life expectancy.
Electrophysiology and nerve biopsy: comparative study in Friedreich's ataxia
and Friedreich's ataxia
phenotype with vitamin E deficiency.
Mary Vida is a 41 year old Florida resident who has been living with Friedreich's ataxia
for 20 years.
The Idis Named Patient Programme we have developed for Santhera will help to ensure that Friedreich's Ataxia
patients with an urgent unmet medical need have access to Catena(R).
Nathan has a condition called Friedreich's Ataxia
- a progressive, genetic disease of the nervous system that has left him using a wheelchair.
Santhera Pharmaceuticals (SWX: SANN, "Santhera"), a Swiss specialty pharmaceutical company with a focus on neuromuscular diseases, and Takeda Pharmaceutical Company Limited (TSE:4502, "Takeda"), jointly announced today that the European Medicines Agency (EMEA) has accepted the filing of the Marketing Authorization Application (MAA) for Santhera's lead compound SNT-MC17 (INN: idebenone, originally developed by Takeda) for the treatment of Friedreich's Ataxia
Angela, from Oxton, Wirral, is hoping for a cure for the neuromuscular disease Friedreich's ataxia
which leaves her unsteady on her feet and with slurred speech.
The 14-year-old, who has the rare degenerative disease Friedreich's Ataxia
, now relies on a wheelchair.
is caused by inadequate production of the protein frataxin which leads to degeneration of the nerves controlling muscle movements.
Edison Pharmaceuticals has previously received Orphan Designation from the FDA for the treatment of inherited respiratory chain diseases of the mitochondria and Friedreich's ataxia
, and from the European Medicines Agency Committee on Orphan Products and Japanese Ministry of Health, Labor and Welfare for the treatment of Leigh syndrome.
She suffers from Friedreich's Ataxia
which affects your coordination and your balance.
Product development, where the Company is preparing to file the Marketing Authorization Approval (MAA) for its lead compound SNT-MC17 (INN: idebenone) for Friedreich's Ataxia
(FRDA) in Europe.
I have got my head around having Friedreich's ataxia
and have had a great life.
Ian suffers from Friedreich's Ataxia
, a progressive inherited disease which usually appears in childhood and attacks the central nervous system.