Discussing the Phase 3 LELANTOS data of FibroGen's Pamrevlumab in patients with non-ambulatory DMDTicker(s): FGEN
Institution: Children's Hospital of Richmond (at VCU)
- Assistant Professor, Division of Child Neurology at VCU & Dual-board certified child neurologist and neuromuscular physician.
- Currently treats 60 to 70 patients with DMD.
- Clinical and research interests are in pediatric neuromuscular disorders with a particular focus on Duchenne Muscular Dystrophy; has served as the PI of several clinical trials in DMD and site investigator for Exon 51.
What are your high level thoughts, including familiarity with this program even before we approached you with this topic?Added By: slingshot_insights
What are personal thoughts on targeting CTGF as just a
general idea in DMD? Are you more optimistic or pessimistic about this pathway making a big
difference in these patients?
Could you talk about the natural history of DMD? For the 46 patients in the placebo
arm what your expectation would be after 104 weeks, what kind of FVC decline
would you expect, and is there even a possibility that the placebo arm would be
Looking at upper limb performance, do you think that
those all correlate together, or what's the chance that we might get a split
result where there's really encouraging signs on one endpoint, but then on the
others maybe not so much?
If this drug did nothing else but stabilize the functional assessment, even
if we don't know the effect size, would that be pretty likely to yield a positive
What is your level of confidence regarding the ambulatory trial, and is the North Star Ambulatory Assessment endpoint more difficult to hit as compared to the non-ambulatory trial
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