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Clinical Challenges: NfL as Emerging Biomarker in Huntington s Disease

Neuron-specific protein might allow for earlier treatment, better prognostication

Daniel claassenFurr stimmingErin furr stimmingVanderbilt university medical centerMcgovern medical schoolResearch utilityDisease integrated staging systemTotal functional capacityHuntington 039s diseaseAntisense oligonucleotideAmyotrophic lateral sclerosis

First-Ever Huntington Staging System May Spur Drug Development

The first-ever staging system for Huntington disease is a key step toward developing drugs that target the disorder before the onset of debilitating symptoms.

United statesNew jerseyErin furr stimmingCristina sampaioMovement disorder societyGreen valley pharmaceuticalsDrug administrationGw pharmaceuticalsHuntington disease society of america centerHuntington disease regulatory science consortiumMcgovern medical schoolPinteon pharmaceuticalsDisease integrated staging systemMedscape medicalDisease regulatory science consortiumIndependence scale

uniQure (QURE) Provides Update on Low-Dose Cohort in Phase I/II Clinical Trial of AMT-130

uniQure (QURE) Provides Update on Low-Dose Cohort in Phase I/II Clinical Trial of AMT-130
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United statesRicardo dolmetschMatt kapustaYear observationsLow dose cohortTotal functional capacityTotal motor scores

uniQure Announces Update on Low-Dose Cohort in Phase I/II

~ Treatment generally well-tolerated with no significant safety issues related to AMT-130 in treated patients through one year of follow-up ~ ~ A mean.

United statesNoord hollandRicardo dolmetschMatt kapustaPhasei ii clinical programNeurofilament light chainYear observationsLow dose cohortTotal functional capacityTotal motor scoresConference callQure conference callNasdaq qureNiqure incMt 130Gene therapy

uniQure Inc : uniQure Announces Update on Low-Dose Cohort in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington s Disease

~ Treatment generally well-tolerated with no significant safety issues related to AMT-130 in treated patients through one year of follow-up ~ ~ A mean reduction of 53.8% of mutant HTT (mHTT) observed

United statesNoord hollandChiara russoRicardo dolmetschMariae cantorMatt kapustaTom malonePhasei ii clinical programNeurofilament light chainYear observationsLow dose cohortTotal functional capacityTotal motor scoresConference callQure forward lookingAnnual report