Neurodegenerative disease and motor neuron disorders
Research activity includes spinal muscular atrophy, amyotrophic lateral sclerosis linked to SOD1 mutation, Parkinson’s disease, and mild cognitive impairment due to Alzheimer’s disease.
- Spinal muscular atrophy
- Amyotrophic lateral sclerosis with SOD1 mutation
- Parkinson’s disease
- Alzheimer’s disease–related cognitive decline
The portfolio also extends to long-term neurological follow-up and treatment persistence in inherited and progressive nervous system disorders.
Multiple sclerosis and central nervous system inflammation
Clinical research covers relapsing forms of multiple sclerosis, including relapsing-remitting multiple sclerosis and pediatric RRMS, with attention to inflammatory activity within the central nervous system.
- Relapsing-remitting multiple sclerosis
- Relapsing forms of multiple sclerosis
- Pediatric multiple sclerosis
- Brain inflammation and MRI-defined disease activity
Work in this area also includes continued evaluation of disease control and longer-term neurological outcomes.
Lupus and cutaneous autoimmune disease
The sponsor funds studies in systemic lupus erythematosus, subacute cutaneous lupus erythematosus, and chronic cutaneous lupus erythematosus, including cases with systemic features and refractory skin disease.
- Systemic lupus erythematosus
- Subacute cutaneous lupus erythematosus
- Chronic cutaneous lupus erythematosus
- Autoimmune skin and systemic manifestations
These studies focus on both skin activity and broader autoimmune involvement in adult populations.
Kidney transplantation and immune-mediated renal disease
Research includes kidney transplant recipients with late antibody-mediated rejection and microvascular inflammation, alongside primary renal autoimmunity such as IgA nephropathy.
- Late antibody-mediated rejection
- Late isolated microvascular inflammation
- IgA nephropathy
- Transplant-related immune injury
The renal portfolio reflects interest in both post-transplant complications and chronic immune-driven kidney disease.
Inherited retinal disease and rare genetic disorders
Additional studies address choroideremia, X-linked retinitis pigmentosa, and Friedreich’s ataxia, highlighting involvement in inherited conditions affecting vision and neurology.
- Choroideremia
- X-linked retinitis pigmentosa
- Friedreich’s ataxia
- Rare genetic disease
These programs include disorders with progressive sensory, retinal, and neurologic impairment.
