Huntington ‘s disease
uniQure Secures FDA Agreement on Accelerated Approval Pathway for Huntington’s Disease Gene Therapy AMT-130
uniQure, FDA, Accelerated Approval, Huntington’s Disease, Gene Therapy, AMT-130, cUHDRS, Neurofilament Light Chain (NfL), Regenerative Medicine Advanced Therapy (RMAT)
Novartis Invests $1 Billion in PTC’s Huntington’s Disease Program
Novartis, PTC, Huntington’s disease, pharmaceutical investment, gene therapy
Novartis Invests $1 Billion in PTC Therapeutics’ Huntington’s Disease Program
Novartis, PTC Therapeutics, Huntington’s disease, Phase 2 drug, global license agreement, collaboration, upfront payment, milestone payments.
Emerging Therapies for Huntington’s Disease: A Review of Promising Treatments
Huntington’s disease, gene therapy, antisense oligonucleotides, stem cell therapy, disease-modifying treatments
Takeda Terminates Partnership with Wave Life Sciences on Huntington’s Disease Program
Takeda, Wave Life Sciences, Huntington’s disease, WVE-003, partnership termination, biotech news
Sage Therapeutics’ Dalzanemdor Fails in Alzheimer’s, Hopes Now Rest on Huntington’s Disease Trial
Sage Therapeutics, Dalzanemdor, Alzheimer’s Disease, Huntington’s Disease, Clinical Trials, Neurodegenerative Disorders
uniQure Announces Positive Interim Data Update Demonstrating Slowing of Disease Progression in Phase I/II Trials of AMT-130 for Huntington’s Disease
uniQure, Huntington’s disease, AMT-130, gene therapy, Phase I/II trials, disease progression, neurofilament light protein (NfL), Unified Huntington’s Disease Rating Scale (cUHDRS), Regenerative Medicine Advanced Therapy (RMAT) designation
UniQure Wins First FDA RMAT in Huntington’s Disease After Strong Phase I/II Data
UniQure, Huntington’s disease, gene therapy, AMT-130, FDA, RMAT designation, Phase I/II clinical trials, neurodegenerative disorder, neurofilament light (NfL), disease progression, composite Unified Huntington’s Disease Rating Scale (cUHDRS).
Wave Life Sciences Achieves Breakthrough in Huntington’s Disease Treatment with WVE-003, Eyes Accelerated Approval
Wave Life Sciences, Huntington’s disease, WVE-003, Phase Ib/IIa study, accelerated approval, mutant huntingtin protein, allele-selective silencing, RNA medicine
PTC Therapeutics Reports Positive Phase II Huntington’s Disease Data, FDA Lifts Partial Clinical Hold
PTC Therapeutics, Huntington’s disease, Phase II data, FDA, partial clinical hold, PTC518, mutant huntingtin protein, clinical trends, safety, tolerability.