30 closest matches · ranked by relevance
X-linked Adrenoleukodystrophy
Primary: Change in Maximal Voluntary Contraction of the Hip Flexors From Baseline to End of Training and to Post-training — 0.94; 0.98; 1.32; 1.37 kg
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Cerebral Adrenoleukodystrophy (CALD)
Primary: Percentage of Participants Who Were Alive and Have None of the 6 Major Functional Disabilities (MFDs) at Month 24 — 85.7 Percentage of participants
Cerebral Adrenoleukodystrophy (CALD)
Primary: Percentage of Participants Who Were Alive and Have None of the 6 Major Functional Disabilities (MFDs) at Month 24 and Without Allo-HSCT or Rescue Cell Administration…
Severe Combined Immunodeficiency
Primary: Number of Participants With Adverse Events — 10; 9; 0; 10 participants
Adenosine Deaminase Deficiency · Severe Combined Immunodeficiencies (SCID)
Primary: Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) — 100; 100; 100; 100 percentage of participants
Ataxia Telangiectasia
Primary: Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS) — 0.94; 2.24 RmICARS score (0-29 points)
Lysosomal Acid Lipase Deficiency
Primary: Percentage Of Participants Achieving Alanine Aminotransferase Normalization — 31; 7; 56; 37 percentage of participants — p=0.0271
Alzheimer's Disease
Primary: Drug-Placebo Difference in Change From Baseline to Week 4 in the Composite Amyloid Burden of the Brain — -0.03; 0.02 SUVr — p=0.22
Leukodystrophy · White Matter Disease · 4H Syndrome
Primary: Changes in Diagnosis Status (Resulting From WGS) — 45 Participants
ADA-SCID
Primary: Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) — 100; 85.71; 100; 92.31 percentage of participants
Retinitis Pigmentosa · X-linked Genetic Diseases
Primary: Rate of LOSS of 31 Hertz Cone Electroretinographic Function — 0.028; 0.022 log microvolts/year — p=0.30
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Smith-Lemli-Opitz Syndrome
Primary: Number of Responders — 18 Participants
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 174; 355; 0; 1 events
Immunologic Deficiency Syndromes
Primary: Survival — 12 Participants
Alzheimer's Disease
Primary: Change From Baseline in Alzheimer's Disease Assessment Scale- Cognitive Subscale (ADAS-Cog11) at 76 Weeks — 7.37; 6.77 units on a scale — p=0.560
Fabry Disease
Primary: Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin — 2; 14; 5; 0 patients
Niemann-Pick Disease, Type C
Primary: Efficacy of Adrabetadex (VTS-270) to Reduce Plasma Levels of a Conjugated Bile Acid, Known as 5α-cholanic Acid-3β, 5α, 6β-triol N-(Carboxymethyl)-Amide — 32.6; 87.9 ng/ml
Alzheimer's Disease
Primary: Change From Baseline in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog11) Score at 76 Weeks — 6.19; 7.29; 7.68 units on a scale — p=0.134
Nervous System Disorder · Genetic Syndrome
Primary: Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population — -2.2; -3.4; -4.0…
Late Infantile Metachromatic Leukodystrophy
Primary: Relative Changes (%) in Gross Motor Function Measurement (GMFM) — -2.98; -5.42; -5.28 Relative % change in total GMFM-88 SOT — p=0.4013
Molybdenum Cofactor Deficiency, Type A
Primary: Overall Survival — 3 Participants
Nervous System Disease · Genetic Syndrome
Primary: Change From Baseline in Modified International Cooperative Ataxia Rating Scale (mICARS) — 0.8; 1.0; 2.3 score on a scale — p=0.0847
Parkinson Disease · Amyotrophic Lateral Sclerosis
Primary: AMDX-2011P Adverse Events Profile — 0; 1; 3 Treatment Emergent Adverse Events TEAEs
Metachromatic Leukodystrophy (MLD)
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 4; 5; 4 participants
HIV Lipodystrophy
Primary: Rate of Total Lipolysis — 0.649; 0.767 mmol FFA/kg/h
Hodgkin Lymphoma
Primary: Complete Response Rate — 34 Participants
Smith-Lemli-Opitz Syndrome
Primary: Hyperactivity Sub-scale of the Aberrant Behavior Checklist-Community (ABC-C). — 8.6; 8.6 units on a scale — p=1.0
Molybdenum Cofactor Deficiency, Type A
Primary: Safety of ORGN001 (Formerly ALXN1101) — 1; 5; 6; 2 events