30 closest matches · ranked by relevance
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…
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
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
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
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
Lysosomal Acid Lipase Deficiency
Primary: Percentage Of Participants Achieving Alanine Aminotransferase Normalization — 31; 7; 56; 37 percentage of participants — p=0.0271
Brain Diseases, Metabolic, Inborn · Urea Cycle Disorder · Ornithine Transcarbamylase Deficiency
Primary: Concentration of Glutamine and Myoinositol by MRS — 4.97; 3.66; 3.78; 4.50 mM — p=0.003
Glucose Transporter Type 1 Deficiency Syndrome · GLUT1 Deficiency Syndrome
Primary: Number of Participants With Reduction in Spike-wave Fraction of the EEG Recording Time — 13 Participants
CDKL5 Deficiency Disorder
Primary: Summary of 28-day Seizure Frequency for Major Motor Seizure Types — 49.17; 54.00; 55.50; 45.03 Seizures per day
Immunologic Deficiency Syndromes
Primary: Survival — 12 Participants
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Primary: Incidence And Severity Of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 13; 5; 7; 7 Participants
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) at 76 Weeks — 7.37; 6.77 units on a scale — p=0.560
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
Infantile Neronal Ceroid Lipofuscinosis
Primary: Change in Cellular Granular Osmiophilic Deposits (GRODs) in Electron Micrographs of Peripheral White Blood Cells. — -0.3317 Average number of GRODs
AL Amyloidosis
Primary: Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation — 4; 3; 6; 13…
Amyotrophic Lateral Sclerosis
Primary: Safety Outcomes: Frequency of Adverse Events — 49; 24; 42 Total Number of Adverse Events — p=0.06
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
Gaucher Disease
Primary: Spleen Volume — 2324.0; 2120.0; 778.0; 1707.7 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
Alzheimer Disease · Dementia
Primary: FCI Score at Follow-up — 147.3; 164.0; 123.3; 150.8 points awarded for correct items
CTX
Primary: Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods — 7.18; 6.96; 7.45; 10.34 natural log of ng/mL — p=<0.0001
Cerebral Amyloid Angiopathy
Primary: Change From Baseline to Day 2 in Cerebrovascular Reactivity as Measured by the Slope (Amplitude Over Time to Peak) From Visual Task-evoked Functional Magnetic Resonance…
Alzheimer Disease · Presbycusis · Lexical Syntactic Disorder
Primary: Link Between Lack of Word and Presbycusis — 38; 38.5; 23; 21.5 score on a scale
Cholesterol Ester Storage Disease(CESD) · Lysosomal Acid Lipase Deficiency · LAL-Deficiency
Primary: Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) — 1; 3; 3; 0 participants
Alzheimer's Disease · Vascular Dementia · Dementia
Primary: Word List Memory Total - ADAS-cog — 14.35; 14.7; 15.1; 14.67 Words recalled
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…