Expert Interview
Diving into the Long-Term Open Label Data for Larimar Therapeutics' Nomlabofusp in Friedreich's Ataxia.
Ticker(s): LRMR, BIIBInstitution: UCLA
- Clinical Professor of Neurology. Director of Ataxia Clinic and Neurogenetics Clinical Trials at UCLA Medical Center.
- Over 30 years of experience diagnosing and treating Friedreich's Ataxia; has served on the National Ataxia Foundation's (NAF) Medical Research Advisory Board, NAF’s Board of Directors and is NAF’s Medical Director.
- Helped develop the National Ataxia Database for clinical research and has participated in 2 natural history studies (for dominant Ataxia and Friedreich’s Ataxia) in addition to numerous clinical drug trials.
How many FA patients do you manage?
Added By: max_adminHow many of your FA patients are on Skyclarys?
Added By: max_adminWhat is your level of concerns regarding the obeserved anaphylaxis risk?
Added By: max_adminWhat is your level of excitement for nomlabofusp on a scale of 1-10?
Added By: max_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: max_adminCan you explain the scientific rationale behind why prior exposure followed by a break increases the chance of anaphylaxis?
Added By: max_adminIf a patient were to receive Lexeo therapeutics gene therapy for FA cardiomyopathy (LX2006), would you also prescribe nomlabofusp?
Added By: max_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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