BioMarin PharmaceuticalBMRN

• Phase 2BMN 351 뒤센 근이영양증 장기 안전성 및 유효성 임상진행 중An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
  0%
• Phase 2보소리티드 저신장 희귀질환 소아 성장 치료 임상모집 중A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
  71%
• Phase 2보소리티드 소아 특발성 저신장증 2상 임상모집 중A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
  71%
• Phase 2BMN 333 소아 연골무형성증 2/3상 임상모집 중A Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
  71%
• Phase 1BMN 351 듀센 근이영양증 치료 1/2상 임상모집 중A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
  55%
• Phase ?팔린직 성인 환자 약물 과민반응 탈감작 치료 임상진행 중Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
  33%
• Phase 23세 미만 연골저형성증 소아 보소리티드 치료 임상모집 중A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
  71%
• Phase ?팔린직 페닐케톤뇨증 환자 대상 장기 안전성 연구모집 중A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
  80%
• Phase ?페닐케톤뇨증 치료제 팔린직 장기 안전성 조사모집 중A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
  83%
• Phase ?팔린직 임신·수유 중 투약 시 태아 및 영아 안전성 연구모집 중A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
  59%
• Phase 3일본 중증 혈우병 A 환자 대상 유효성·안전성 3상 임상진행 중Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
  59%
• Phase ?팔린직 투여 성인 환자 면역 조절 분석 임상시작 전Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
  98%

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