Expert Interview
A Second View: Diving into the Long-Term Open Label Data for Larimar Therapeutics' Nomlabofusp in Friedreich's Ataxia.
Ticker(s): LRMR, BIIBInstitution: SUNY Downstate Medical Center
- Director of the Muscular Dystrophy Association Clinic and Associate Professor of Neurology at SUNY Downstate Medical Center.
- Treats 8 patients with ATTR-PN, 15 patients for Friedreich’s Ataxia and 90 patients with myasthenia gravis.
- Research Interests in HIV-related neuropathy, inflammatory myopathies and has a clinical specialty in Neuromuscular disease.
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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