HDTrialFinder – Find Huntington's disease Trials in Europe

A Rollercoaster Ride to Hope

7. July 20268. July 2026 Claudia AzañedoUkategorisert

A Rollercoaster Ride to Hope The AMT-130 Story, and What the FDA’s June 2026 Announcement Means for Our Community Our community’s first meeting with the pharmaceutical company uniQure took place in September 2018. At the time, the idea that a…

AN UPSIDE DOWN APPROACH TO CLINICAL TRIALS

19. June 202626. June 2026 Claudia AzañedoUkategorisert

SKYHAWK THERAPEUTICS AN UPSIDE DOWN APPROACH TO CLINICAL TRIALS: LEADING TO SOME EXCITING RESULTS Skyhawk Therapeutics, a US-based pharmaceutical company, has developed a new medication that aims to slow the progression of Huntington’s Disease. Let’s start from the beginning and…

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siRNA

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phenoptype

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oxidative seres

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Metabolism & bioenergetics

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Small Molecule

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Nucleic acid

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SNP-single nucleotide polymorphisms

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at risk

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TFC-total functional capacity

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Double-blinded

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Open label

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Gene therapy

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UHDRS- Unified Huntington Disease Rating Scale

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CAG repeat

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Wild-type

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Tolerabilty

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Striatum

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Randomized allocation

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Radioligand

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Protein

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Premanifest / Prodromal

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Placebo

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PK - Pharmacokinetics

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PD - Pharmacodynamics

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PET scan

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Neuron

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MRI

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mHTT

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Manifest

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Longitudinal study

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HTT

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fMRI

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CSF - cerebrospinal fluid

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Efficacy

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ASO(Antisense oligonucleotides)

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Biomarker

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BDNF

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Allele

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Plasma

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Gene