Huntington ‘s disease
uniQure Faces Delay as FDA Walks Back Accelerated Approval for Huntington’s Gene Therapy
uniQure; AMT-130; Huntington’s disease; gene therapy; FDA; accelerated approval; regulatory setback; clinical trial
uniQure’s Gene Therapy AMT-130 Significantly Slows Huntington’s Progression in Pivotal Trial
Gene therapy; uniQure; AMT-130; Huntington’s disease; clinical trial; disease progression; FDA approval; huntingtin-lowering; neurodegeneration
PTC’s Novartis-partnered Huntington’s drug disappoints in mid-stage data reveal
PTC518, factor IX, Huntington ‘s disease, Market
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