Expert Interview
A Third View: Diving into the Long-Term Open Label Data for Larimar Therapeutics' Nomlabofusp in Friedreich's Ataxia
Ticker(s): LRMR, BIIBInstitution: Children's Hospital of Philidelphia
- Attending neurologist in the Division of Neurology at Children's Hospital of Philadelphia and Director of the Friedreich's Ataxia Program.
- Research focuses on NMDA & Glutamate receptors.
- Maintains a dynamic program that involves clinical, translational, and basic science research efforts focused on Friedreich ataxia.
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: wilson_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: wilson_adminCan you explain the scientific rationale behind why prior exposure followed by a break increases the chance of anaphylaxis?
Added By: wilson_adminIf a patient were to receive Lexeo therapeutics gene therapy for FA cardiomyopathy (LX2006), would you also prescribe nomlabofusp?
Added By: wilson_adminFor 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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