The Unyielding Unmet Need in Huntington’s: Why Pipeline Progress is Redefining Therapeutic Strategy
Gene therapies, shifting diagnosis paradigms, and a new calculus for patient eligibility are changing how companies must approach Huntington’s disease Huntington’s disease (HD) has long been recognized for its [...]
Why Tissue Type Matters Most in the Next Phase of DM1 Therapeutics
How multisystem involvement, patient heterogeneity, and new modalities are redefining the DM1 opportunity for drug developers Myotonic dystrophy type 1 (DM1) stands apart on the rare neuromuscular landscape as [...]
Why IgA Nephropathy Is Entering a New Commercial Phase
What evolving risk definitions, shifting treatment expectations, and B cell immunomodulation mean for drug developers IgA nephropathy (IgAN) is no longer a niche renal indication defined by slow progress and [...]
MARKETVUE® REPORT: More than half of STXBP1 Encephalopathy patients experience refractory seizures with currently available antiepileptics
Despite significant unmet need, there are currently no disease-modifying therapies available for the treatment of STXBP1 encephalopathy, although there are at least six assets in preclinical development, according to findings [...]
The Unyielding Unmet Need in Huntington’s: Why Pipeline Progress is Redefining Therapeutic Strategy
Gene therapies, shifting diagnosis paradigms, and a new calculus for patient eligibility are changing how companies must approach Huntington’s disease Huntington’s disease (HD) has long been recognized for its [...]
Why Tissue Type Matters Most in the Next Phase of DM1 Therapeutics
How multisystem involvement, patient heterogeneity, and new modalities are redefining the DM1 opportunity for drug developers Myotonic dystrophy type 1 (DM1) stands apart on the rare neuromuscular landscape as [...]
Why IgA Nephropathy Is Entering a New Commercial Phase
What evolving risk definitions, shifting treatment expectations, and B cell immunomodulation mean for drug developers IgA nephropathy (IgAN) is no longer a niche renal indication defined by slow progress and [...]
MARKETVUE® REPORT: More than half of STXBP1 Encephalopathy patients experience refractory seizures with currently available antiepileptics
Despite significant unmet need, there are currently no disease-modifying therapies available for the treatment of STXBP1 encephalopathy, although there are at least six assets in preclinical development, according to findings [...]