Chanarin-Dorfman Syndrome - 6 Studies Found

Completed : International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases
: Neutral Lipid Storage Disease
: 2016-08-04
Completed : The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM)
: Neutral Lipid Storage Disease
: 2012-01-16
: Drug: Fibrate treatment Patients will receive a dosage of 400mg Bezafibrate every day during 28 weeks
Completed : Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M
:
  • Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV)
  • Neutral Lipi
    : 2016-07-06
    : Dietary Supplement: CNT-02 Each subject will take 2.0g of the investigational product orally 3 times a d
Active, not recruiting : Clinical Trial in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
: Lysosomal Acid Lipase Deficiency
: 2014-07-07
: Drug: sebelipase alfa Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). The investig
Terminated : Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102
:
  • Lysosomal Acid Lipase Deficiency
  • Wolman Disease

: 2011-11-10
: Drug: SBC-102 SBC-102 is a recombinant human lysosomal acid lipase (rhLAL). The investigational medicina
Active, not recruiting : SurVival of Lysosomal Acid Lipase Deficiency (LAL-D) Infants Treated With SebelipAse aLfa
:
  • Lysosomal Acid Lipase Deficiency
  • Wolman Disease

: 2011-06-09
: Drug: Sebelipase alfa (SBC-102) Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). Th