The interim data from the AMT-130 trial demonstrated an 80% slowing of disease progression in the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 24 months for high-dose patients. Can you elaborate on the significance of these findings for the HD community?

The study showed a statistically significant reduction of cerebrospinal fluid (CSF) neurofilament light protein (NfL) levels in patients treated with AMT-130. How does the reduction of NfL correlate with the slowing of neurodegeneration in HD?

Given that AMT-130 showed near-baseline stability in motor and cognitive function over 24 months, what potential long-term benefits can patients expect from this treatment?

AMT-130 has been generally well-tolerated with a manageable safety profile. Can you discuss any specific adverse events observed and how they were managed?

The PIVOT-HD study indicated dose-dependent lowering of mutant huntingtin (mHTT) protein in both blood and CSF. What implications does this have for dose optimization in future treatments?

PTC518 showed favorable trends on HD clinical assessments, including the Total Motor Score (TMS). How do these trends translate into real-world improvements in daily functioning for HD patients?

With uniQure's AMT-130 receiving the first-ever Regenerative Medicine Advanced Therapy (RMAT) designation for HD and the FDA lifting the partial clinical hold on PTC518, what are the next regulatory steps for these therapies?

How do the clinical and biomarker outcomes of AMT-130 compare to those observed with PTC518, and what does this mean for future treatment strategies in HD?

Uniqure has not released data on reduction of HTT for their trial. Why is that?

Does PTC518 lower the EXON1 protein like AMT130 is designed to do?

There are several different endpoints that are being used by companies to measure the efficacy of their therapies including, cUHDRS score, caudate volume, mutant Huntington, motor scores and NFL. Which of these endpoints best represent the disease and progression? Additionally, could you think of a reason why the FDA asks and accepts these different clinical endpoints?

Recently, we have seen announcements of siRNAs being able to penetrate the brain via subq and IV infusion. Do you think patients will wait for such therapies to be approved rather than having a one-and-done gene therapy albeit with a surgery? Considering the disease progression, I can imagine patients do not wish to wait for other treatments. In line with this, would patients prefer daily oral over a one-time treatment?