4 results
Approved WMORecruiting
To evaluate the long-term safety and tolerability of ARGX-117 in adult participants with MMN
Approved WMORecruiting
Rationale:Multifocal motor neuropathy (MMN) is a rare neuropathy characterized by progressive asymmetric weakness and atrophy without sensory abnormalities. MMN is considered a chronic immune-mediated neuropathy driven by the classical complement…
Approved WMORecruiting
The primary objective of this study is to evaluate the efficacy of olipudase alfa in adult patients with acid sphingomyelinase deficiency.
Approved WMORecruiting
Our study objective is to evaluate the efficacy of acetazolamide for the treatment of cystoid macula edema in inherited retinal dystrophies in anticipation of future clinical trials.