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This is an open-label pilot study of human leukocyte antigen (HLA)-matched related and unrelated donor hematopoietic stem cell (HSC) transplant (also referred to as peripheral blood stem cell [PBSC] transplant or bone marrow transplant [BMT]) for up to 20 patients with severe combined immune
Friedreich ataxia (FRDA) is a devastating neurodegenerative disease that affects children and young adults. Patients become progressively unable to coordinate movements and walking until severe disability ensues. Most patients develop dilated cardiomyopathy and congestive heart failure. The disease
Participants will be assigned to receive the full immunosuppressive therapy indicated to treat chronic ABMR (including plasmapheresis (PF) 7x, intravenous immune globulins 100 mg/kg 7x, corticosteroid) and MSC infusions (2x106cells/kg bw after PF) (Group 1) and be compared to historic controls
AGHD is widely recognized as a specific clinical entity and recombinant human growth hormone (r-hGH) therapy has become a standard practice since 90s thanks to its beneficial effects on body composition, muscle strength, bone mass, cardiovascular risk and patients' exercise capacity and QoL. All the
INTRODUCTION
Overweight and obesity represent a risk factor for hormone dependent cancers such as breast, endometrium, ovarian, uterine and gastrointestinal cancer.
In particular, overweight and obesity are risk factors for breast cancer in post-menopause women since they are associated to higher
Phosphodiesterase-5 (PDE5) inhibitors are currently used in the therapy of erectile dysfunction (ED), which is considered to be an early symptom of cardiovascular disease (CD). Impairment of endothelial function is the early lesion preceding atherosclerosis and is common to ED, CD and vascular
Acute rejection after kidney transplantation can lead to graft loss by irreversible acute rejection or to interstitial fibrosis/ tubular atrophy that can induce graft loss. Induction therapy by either T-cell depleting polyclonal antibodies such as Anti-Thymocyte Globulins (ATG) or non-depleting
Autoimmune hepatitis (AIH) is an immune-mediated necroinflammatory disease of the liver characterized by elevation of IgG, presence of characteristic autoantibodies, and histological feature of interface hepatitis. Standard therapy consists of a combination of corticosteroids and azathioprine, which
A. Primary Objectives:
1. The percentage of patients who develop chronic allograft injury (CAI) progression during the first 12 months post-transplant protocol biopsy (i.e., higher grade of IF/TA at either the 6 or 12 month protocol biopsy in comparison with the baseline biopsy).
2. The incidence
Sterile osseous defects of the knee joint are temporarily stabilised with a hinge arthroplasty or an intramedullary nail in the case of a long femoral defect.
Assisted passive motion and isometric exercises are possible and necessary to avoid contractions and muscular atrophy during waiting time.
Purpose: The purpose of this comparison is to determine the best of two protein sources in the embryo culture media for future use in the Presbyterian Assisted Reproductive Technology Services (ARTS) Program.
Background: For embryos to grow in the incubator, a protein source is essential in the
This is an open-label pilot study of HLA-matched allogneic and matched unrelated donor (MUD) hematopoietic stem cell (HSC) transplant (also referred to as peripheral blood stem cell (PBSC) or bone marrow transplant (BMT)) for patients with X-linked severe combined immune deficiency (XSCID). XSCID is
To evaluate the efficacy of two treatment modalities: pulse cyclophosphamide versus high dose cyclophosphamide and anti-thymocyte globulin (ATG) rescued with autologous peripheral blood stem cell transplantation (PBSCT). The primary endpoints to be considered in this study are:
I)Time to Treatment
To assess the efficacy of autologous PBSCT versus FDA approved standard of care ( i.e. interferon, glatiramer acetate, mitoxantrone, natalizumab, fingolimod, or tecfidera) for inflammatory multiple sclerosis (MS) failing failing alternate approved therapy. The endpoints to be considered in this
Chronic Focal Encephalitis (Rasmussen's Encephalitis) is a condition characterized by a progressive hemiparesis, cognitive decline (including loss of language skills if the language dominant hemisphere is involved) and epileptic seizures that are typically refractory to medical treatment