Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich's ataxia (a pilot study) - ND Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in ...
Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich's ataxia (a pilot study) - ND Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in ...
Friedreich ataxia (FRDA) is a rare autosomal recessive neurodegenerative disorder caused a mutation in the FXN gene, which encodes a protein named frataxin. As a result of the mutation, frataxin is quantitatively reduced but qualitatively normal. Thus, any pharmacological agent able to increase frataxin intracellular levels would have a great therapeutic relevance. MedDRA version: 6.1;Level: PT;Classification code 10003591 Friedreich ataxia (FRDA) is a rare autosomal recessive neurodegenerative disorder caused a mutation ...