Recently the intrathecal administration has been proposed as part of a hopeful therapy for FA (Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich’s ataxia). This paper shows (in laboratory animals) the feasibility of the use of intrathecal administration to reach efficiently the dorsal root ganglia.
This presentation explains the current state of the art in other neurological diseases in which it is required reach the CNS.
Mission: Improve outcomes for epileptic patients who don’t respond to conventional treatments by administering reformulated, micro-doses of anti-epileptic drugs directly to the brain.
Tuesday, February 23, 2016
Retrotope Advances RT001 in Clinical Trials to Treat Friedreich's ataxia
(Ref: Marketwired) February 22nd, 2016
LOS ALTOS, CA--(Marketwired) - Retrotope, a privately held clinical stage pharmaceutical company, today announced the successful completion of the first dose cohort and the opening of patient enrollment for the highest dose cohort in its ongoing 28-day study of orally dosed RT001 in Friedreich's ataxia (FA) patients. RT001 was well tolerated and no serious adverse events or dose limiting toxicities were observed.
LOS ALTOS, CA--(Marketwired) - Retrotope, a privately held clinical stage pharmaceutical company, today announced the successful completion of the first dose cohort and the opening of patient enrollment for the highest dose cohort in its ongoing 28-day study of orally dosed RT001 in Friedreich's ataxia (FA) patients. RT001 was well tolerated and no serious adverse events or dose limiting toxicities were observed.
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