3 results
The primary objective of this study is to assess the efficacy of Ecopipam to reduce self-injurious behaviors (SIB) in adults and children with Lesch-Nyhan Disease (LND) in an outpatient setting. The secondary objectives of this study are to assess…
The primary objective of this study is to evaluate the safety and effectiveness of the Genous Bio-engineered R stentTM in conjunction with optimal statin therapy (80mg of atorvastatin), in the treatment of elective patients with up to two de novo…
* To decrease mortality in MS-LCH by an early switch of patients with risk organ involvement, who do not respond to front-line therapy, to a more intensive treatment (Stratum III or Stratum IV).* To reduce reactivation rates and permanent…