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Enroll -HD: A Prospective Registry Study in a Global Huntington's Disease Cohort

The motor section of the UHDRS assesses motor features of HD with standardized ratings of oculomotor function, dysarthria, chorea, dystonia, gait, and postural stability. UHDRS 99 Motor, UHDRS Diagnostic Confidence Level.

Recruiting

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Prenatal presentation of symptoms (e.g. hydrops) appearing on fetal imaging such as ultrasound and ECHO.

Recruiting

MOON Shoulder Instability-Cohort of Patients Undergoing Operative Treatment.

Patients will fill out PRO's after surgery

Recruiting

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (C...

The primary objective of this access and distribution protocol is to examine the incidence of neutrophil recovery of ≥500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not FDA licensed.

Recruiting

P-BCMA-ALLO1 Allogeneic CAR-T Cells in the Treatment of Subjects With Multiple Myeloma

Rate of dose limiting toxicities (DLT)

Recruiting

DB107-RRV, DB107-FC, and Radiation Therapy With or Without Temozolomide (TMZ) for High Grade Gl...

Tolerability is defined as the proportion of participants receiving at least one dose of DB107-RRV and DB107-FC with a reported dose-limiting toxicity for all participants in Phase I.

Recruiting

P-MUC1C-ALLO1 Allogeneic CAR-T Cells in the Treatment of Subjects With Advanced or Metastatic S...

Number of subjects with a dose limiting toxicity (DLT)

Recruiting

Autologous Gene Therapy for Artemis-Deficient SCID

Patient survival status and (if applicable) cause of death will be recorded to assess overall survival.

Recruiting

MammaPrint, BluePrint, and Full-genome Data Linked With Clinical Data to Evaluate New Gene EXpr...

Create a large scale registry of full genome expression data with clinical data to investigate possible new gene associations with prognostic or predictive value.

Recruiting

MammaPrint, BluePrint, and Full-genome Data Linked With Clinical Data to Evaluate New Gene EXpr...

Create a large scale registry of full genome expression data with clinical data to investigate possible new gene associations with prognostic or predictive value.

Recruiting