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…
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
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
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
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Immunologic Deficiency Syndromes
Primary: Survival — 12 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
Smith-Lemli-Opitz Syndrome
Primary: Number of Responders — 18 Participants
Alport Syndrome
Primary: Number of Participants With Adverse Events Associated With Administration of 0.75 mg/kg of ELX-02 Once Daily — 3 Participants
Lysosomal Acid Lipase Deficiency
Primary: Percentage Of Participants Achieving Alanine Aminotransferase Normalization — 31; 7; 56; 37 percentage of participants — p=0.0271
Wilson Disease
Primary: Percentage Of Participants With Normalized Concentrations Of NCC — 85.7 percentage of participants
Klinefelter Syndrome · XXY Syndrome
Primary: Change From Baseline on the Delis-Kaplan Executive Function System — -0.72; 1.29; 1.28; 0.65 Scaled scores
Molybdenum Cofactor Deficiency, Type A
Primary: Overall Survival — 3 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
Sjogren-Larsson Syndrome
Primary: Visual Index Ichthyosis Severity (VIIS) Scaling Score as Assessed by the Investigator in ADX-102 Treated Patients — -0.7 units on a scale
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
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: Safety of ORGN001 (Formerly ALXN1101) — 1; 5; 6; 2 events
Smith-Lemli-Opitz Syndrome
Primary: Change in Plasma Cholesterol — 37.8 change (%) compared to baseline — p=0.011
X-linked Hypophosphatemia · Hypophosphatemic Rickets · Hypophosphatemic Rickets, X-Linked Dominant
Primary: Change in Serum Phosphate in Adults and Children With XLH Between Baseline and 12 Months — 2.6; 2.1 mg/dl — p=0.318
XMEN
Primary: Participants With a ≥0.5 Log Reduction in the Number of EBV-infected B Cells After Magnesium Supplementation as Compared to Placebo - Phase 1 — 0; 0 Participants
Hodgkin Lymphoma
Primary: Complete Response Rate — 34 Participants
Alzheimer Disease
Primary: Number of Participants With Treatment-Emergent Adverse Event (TEAEs) — 34; 26 Participants
Ataxia Telangiectasia
Primary: Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS) — 0.94; 2.24 RmICARS score (0-29 points)
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
Wilson Disease
Primary: Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period) — 92.8 μg/g — p=0.1002