Expert Interview
Discussing Larimar Therapeutics Long-Term Open Label Data Update for Nomlabofusp in Friedreich's Ataxia
Ticker(s): LRMR, BIIBInstitution: University of Louisville
- Professor of Neurology, Medical Geneticist, Endowed Chair of Parkinson’s Disease Research, & Director, Movement Disorders Program at University of Louisville.
- Manages 90 patients with Huntington's disease and 35 patients with Friedreich’s Ataxia.
- Research interests are focused on understanding the underlying genetic basis of hereditary spastic paraplegia and essential tremor; clinical interests include surgical therapies for Parkinson's disease, essential tremor and dystonia, and botulinum toxin procedures for dystonia and spasticity.
How many FA patients do you manage?
Added By: wilson_adminHow many of your FA patients are on Skyclarys?
Added By: wilson_adminWhat is your level of concerns regarding the obeserved anaphylaxis risk?
Added By: wilson_adminWhat is your level of excitement for nomlabofusp on a scale of 1-10?
Added By: ben_adminGiven the protocol, there is a good chance of black box and REMS, how would this impact your willingness to prescribe based on the data set so far?
Added By: jordan.opieCan you explain the scientific rationale behind why prior exposure followed by a break increases the chance of anaphylaxis?
Added By: jordan.opieIf a patient were to receive Lexeo therapeutics gene therapy for FA cardiomyopathy (LX2006), would you also prescribe nomlabofusp?
Added By: jordan.opieFor LRMR, what minimum mFARS effect (e.g., ≥2–3-point advantage or ≥40–50% slowing) over what duration (≥12–24 months) would convince you the drug is clinically efficacious?
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