dm+d

Unassigned

New Medicines

Amyotrophic lateral sclerosis (motor neurone disease) with Fused in Sarcoma Mutations (FUS-ALS)

Information

New molecular entity
Ionis Pharmaceuticals
Ionis Pharmaceuticals

Development and Regulatory status

None
None
Phase III Clinical Trials

Category

Prevents production of mutant variation of the Fused in Sarcoma (FUS) protein, which is responsible for motor neuron degeneration
ALS is the most common form of motor neurone disease; annual incidence of about 2 cases per 100,000 population and prevalence is about 5-7 per 100,000 [1]
Amyotrophic lateral sclerosis (motor neurone disease) with Fused in Sarcoma Mutations (FUS-ALS)
Intrathecal