Deficiency drug bone marrow transplantation. Medical search. Frequent questions

The disorder occurred in the ninth patient two months after allogeneic bone marrow transplantation. (nih.gov)

Dr. Bishop will discuss current research efforts that focus on increasing the application of stem cell transplantation to a broader patient population by increasing the potential stem cell donor pool, decreasing transplant-related toxicities and investigating its use in diseases not commonly treated with stem cell transplantation. (nih.gov)
Plasma ammonium levels were measured in patients on the acute leukemia service or on the bone marrow transplant service at The Johns Hopkins Hospital, and a level more than twice normal was considered diagnostic of hyperammonemia. (nih.gov)
When primary immunodeficiency is likely to cause significant harm or death, an allogeneic hematopoietic stem cell transplant (HSCT), more commonly known as a bone marrow transplant (BMT), may be the best treatment option. (primaryimmune.org)
Since only a small number of stem cells is released into the blood stream, a cell growth stimulating drug is administered to donors prior to the donation to dramatically increase the volume of stem cells in the blood for collection and transplant. (blood.ca)
To prepare for a transplant, the recipient is usually given high doses of radiation, chemotherapy or both to destroy the diseased marrow. (blood.ca)
People who receive unrelated stem cell transplants must stay isolated in a sterile room until the transplant is successful and the new bone marrow has produced enough white blood cells to protect against bacteria. (blood.ca)
Racial and Socioeconomic Disparities in Long-Term Outcomes in =1 Year Allogeneic Hematopoietic Cell Transplantation Survivors: A Center for International Blood and Marrow Transplant Research Analysis. (dana-farber.org)
According to Reference 1, the only cure for CML is a transplant of stem cells, usually from bone marrow. (empowher.com)
For example, if it is a malignancy, we are thinking of doing a transplant, the need to do a bone marrow examination. (atheneum.ai)
Current management strategies include prophylactic penicillin and immunizations to decrease the occurrence of pneumococcal infections, hydroxyurea (a disease-modifying agent), blood transfusions (for symptomatic acute anemia, stroke management, preoperative optimization), and bone marrow transplant. (ccjm.org)

Today, bone marrow transplantation is more commonly referred to as stem cell transplantation, as stem cells can be obtained from several other sources other than the bone marrow, including the peripheral blood and umbilical cord blood. (nih.gov)
Hematopoietic stem cells may be collected from the bone marrow, the peripheral blood, or from umbilical cord blood, so HSCT procedures may be called bone marrow transplants (BMT), peripheral blood stem cell transplants, or cord blood transplants depending on the source of the stem cells. (primaryimmune.org)
They come from bone marrow, circulating (peripheral) blood or umbilical cord blood . (blood.ca)

Although hematopoietic stem cell transplantation remains the mainstay of treatment for ADA-deficient SCID, 2 other treatment options are available, namely enzyme replacement therapy with PEG-ADA and autologous hematopoietic stem cell gene therapy. (nih.gov)
Hematopoietic Stem Cell Transplantation Hematopoietic stem cell (HSC) transplantation is a rapidly evolving technique that offers a potential cure for hematologic cancers ( leukemias, lymphomas, myeloma) and other hematologic disorders. (msdmanuals.com)
He conducted research on hematopoietic stem cell biology, bone marrow transplantation, immune tolerance and treatment strategies for a mouse model of human enzyme deficiency. (biocomcro.org)
Akari has been granted Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA for nomacopan for the treatment of pediatric HSCT-TMA and orphan drug designation from the European Commission for treatment in hematopoietic stem cell transplantation. (woodtv.com)
Hematopoietic stem cell transplantation (HSCT) is a medically complex modality that can offer a chance of cure to children with both malignant and nonmalignant conditions. (dana-farber.org)
Survivors of Pediatric Hematopoietic Stem Cell Transplantation Exhibit Progressive Diastolic Dysfunction Over Years of Follow-Up. (dana-farber.org)
Sickle cell anemia, thalassemia, and glucose-6-phosphate dehydrogenase (G6PD) deficiency - as well as the therapeutic modalities of chemotherapy , radiation therapy , and hematopoietic stem cell transplantation (which includes bone marrow and blood stem cell transplantation ) for aplastic anemia - are addressed in detail in separate fact sheets. (cdho.org)
or chemotherapy and/or radiation therapy [as a prelude to hematopoietic stem cell transplantation ] in the management of sickle cell disease or aplastic anemia). (cdho.org)

The goal of this Funding Opportunity Announcement (FOA) is to solicit applications on lysosomal storage disorders (LSDs) focused on improving CNS treatment outcomes, enhancing the effectiveness of delivery and targeting of cells, enzymes, drugs and genes into the brain, and developing novel therapeutic modalities, such as implantable biocapsules and micro-electro-mechanical systems (MEMS)-based devices. (nih.gov)
Lysosomal storage disorders constitute a group of recessive genetic diseases resulting from cellular enzymatic deficiencies of acid hydrolases that normally catalyze the metabolism of glycoproteins, glycolipids and other macromolecules, or from defects in transporter proteins leading to pathogenic accumulation of these substances in lysosomes. (nih.gov)
Stem cell transplantation is commonly used for a variety of malignant disorders such as leukemias and lymphomas. (nih.gov)
However, it is also used for a number of other diseases such as immune deficiency states and non-malignant blood disorders. (nih.gov)
Dietary restriction has shown promise for disorders such as lysosomal acid lipase deficiency (Wolman disease), as has incorporation of lipid-lowering drugs in the regimen along with sebelipase alpha, a recombinant enzyme replacement therapy. (medscape.com)
Niemann-Pick disease is a group of autosomal recessive disorders caused by an accumulation of fat and cholesterol in cells of the liver, spleen, bone marrow, lungs, and, in some instances, brain. (nih.gov)
ADA and PNP deficiencies are autosomal recessive disorders. (medscape.com)
In both disorders, the enzyme deficiencies result in accumulation of toxic metabolites, especially in lymphocytes. (medscape.com)
In both metabolic disorders, the enzyme deficiencies cause the accumulation of metabolites that are toxic to lymphoid lineage cells. (medscape.com)
Impact of cytoreduction and remission status on hematopoietic cell transplantation outcomes in pediatric myelodysplastic syndrome and related disorders. (dana-farber.org)
Using a functional classification scheme, neutropenia of childhood may be categorized as either intrinsic disorders of myeloid production and proliferation or acquired disorders resulting from extrinsic factors, such as infections, drugs, or immune-mediated mechanisms. (musculoskeletalkey.com)

Caused by deficiencies such as the human immunodeficiency virus, acquired immunodeficiency virus and cancers, which warrant immunosuppression during chemotherapy, immunodeficiency renders the patient vulnerable to viruses, infections, and diseases that, in the immunocompromised state, could develop lethally. (ijpc.com)
Severe immunodeficiency, including those who are undergoing bone marrow transplantation or those with primary or acquired immunodeficiency who require isolation. (clinicaladvisor.com)

Dr. Zheng's clinical interests include leukemia, bone marrow transplantation, immunology and host defenses. (childrens.com)

In purine nucleoside phosphorylase deficiency, similar changes occur, resulting in elevated deoxy-GTP (dGTP) levels. (medscape.com)
In a further description of the mechanism of T-cell depletion in purine nucleoside phosphorylase deficiency, Arpaia et al reported increased in vivo apoptosis of T cells and increased in vitro sensitivity to gamma irradiation in a murine model. (medscape.com)
[ 4 ] The immune deficiency in purine nucleoside phosphorylase deficiency may be the result of inhibited mitochondrial DNA repair due to the accumulation of dGTP in the mitochondria. (medscape.com)

Liver Transplantation Liver transplantation is the 2nd most common type of solid organ transplantation. (msdmanuals.com)
Almost always, transplantation is done to replace or restore the function of an end-stage diseased organ, thereby restoring an essential function and improving patient survival. (msdmanuals.com)
New and higher modality treatments like immunotherapy, targeted therapy, biological therapy, organ or bone marrow transplantation have always been out of the reach because of enormous economic burden. (waocp.com)

Transfer of the ADA gene into bone marrow cells and peripheral blood lymphocytes for the treatment of patients affected by ADA-deficient SCID. (nih.gov)
Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with CD34+ selected autologous peripheral blood cells transduced with a human ADA gene. (nih.gov)
ADAGEN ® (pegademase bovine) Injection is a modified enzyme used for enzyme replacement therapy for the treatment of severe combined immunodeficiency disease (SCID) associated with a deficiency of adenosine deaminase. (nih.gov)
Severe combined immunodeficiency disease (SCID) associated with a deficiency of ADA is a rare, inherited, and often fatal disease. (nih.gov)
The pharmacokinetics and biochemical effects of ADAGEN ® (pegademase bovine) Injection have been studied in six children ranging in age from 6 weeks to 12 years with SCID associated with ADA deficiency. (nih.gov)
Purine nueoside phosphorylase (PNP) deficiency causes a form of severe combined immunodeficiency (SCID) characterized by profound T cell deficiency, failure to thrive (FTT), recurrent deep seeded infection, developmental delay, progressive neurological deterioration, and autoimmune complications. (medscape.com)
However, one of the expected difficulties is that PNP deficiency can be missed with current newborn SCID screening measures. (medscape.com)
ADA deficiency results in absence of T, B, and NK cells, resulting in a SCID with marked lymphopenia. (medscape.com)
ADA deficiency causes a SCID that accounts for approximately 20% of all SCID cases. (medscape.com)

One possible cause of this mysterious hemolysis might be a glucose-6-phosphate dehydrogenase (G6PD) deficiency (G6PDD). (perfusiontheory.com)

The one long-term survivor is currently alive and well without neurologic sequelae 250 days after autologous bone marrow transplantation. (nih.gov)
Vimal is comfortable sharing his experiences with both autologous and allogenic stem cell transplantation practices in India. (atheneum.ai)

Class I patients have a severe deficiency and chronic hemolytic anemia. (perfusiontheory.com)

San Diego is a major oncology research center and we have CROs that can advance oncology drug programs from concept all the way through Phase 2 and 3 clinical trials. (biocomcro.org)

Three-drug anti-tuberculous therapy (with rifampin, isoniazid, and streptomycin) was introduced despite chest and bone radiographs that were negative for infection, no abnormalities found on funduscopy, and negative results of Ziehl-Neelsen staining of lymph node tissue. (cdc.gov)
Improvement in immune function and diminished frequency of opportunistic infections compared with the natural history of combined immunodeficiency due to ADA deficiency only occurs after metabolic abnormalities are corrected. (nih.gov)

Background Early detection of pulmonary morbidity following haematopoietic stem cell transplantation (HSCT) remains an important challenge for intervention, primarily due to the insensitivity of spirometry to early change, and in paediatrics, patient compliance provides additional challenges. (ersjournals.com)

In adenosine deaminase deficiency, adenosine and adenine accumulate in the plasma. (medscape.com)

They tended to develop in patients who had a type of cancer that suppressed their cellular immune function as in chronic lymphocytic leukemia, lymphoma, Hodgkin's or after bone marrow transplantation. (healthworkscollective.com)
One of the lines of evidence is, first of all, in HIV, in the AIDS realm where the HIV or AIDS virus actually results in depletion of T cell populations and these patients are at increased risk for developing cancers including blood cancers like lymphoma as a result of the immune deficiency that results. (healthtree.org)

Risk of these complications is minimized by pretransplantation screening and immunosuppressive therapy during and after transplantation. (msdmanuals.com)
There are drugs to ablate the bone marrow or destroy the bone marrow and combination of immunosuppressive drugs, which help in preventing graft failure. (atheneum.ai)

The World Health Organization classifies G6PDD variants according to the magnitude of the enzyme deficiency and the severity of hemolysis (Table 1). (perfusiontheory.com)

The clinical effects of ADA deficiency are manifest most dramatically in the immune system, where it leads to severe lymphopenia. (nih.gov)
Bone marrow transplantation has been in clinical use for more than 30 years. (nih.gov)
Although individuals with the lowest factor VII levels are most likely to be symptomatic, patients with identical mutations may have marked differences in clinical bleeding, suggesting that other factors may contribute to the clinical manifestations of factor VII deficiency. (medscape.com)
In 2017, the US Food and Drug Administration (FDA) approved L-glutamine oral powder for reducing acute complications of SCD, and many other drugs are in development and undergoing clinical testing. (ccjm.org)

Over time, excessive storage of fats can cause permanent cellular and tissue damage, particularly in the brain, peripheral nervous system (the nerves from the spinal cord to the rest of the body), liver, spleen, and bone marrow. (nih.gov)
Registered adult stem cell donors may be asked to donate either peripheral blood or bone marrow stem cells depending on the physician's choice of therapy for the patient. (blood.ca)
We ask donors to donate stem cells from their peripheral blood, bone marrow or cord blood. (blood.ca)
Where bone marrow donation is a surgical procedure, peripheral blood is a non-surgical procedure done in an outpatient clinic. (blood.ca)

Cyclic oscillations in bone marrow function result in neutropenia with nadirs at intervals of 19 to 21 days. (musculoskeletalkey.com)
Bone marrow examinations during periods of neutropenia may show granulocytic hypoplasia or an apparent arrest of maturation. (musculoskeletalkey.com)

Conventional treatment is based on transplantation of HLA-identical bone marrow from siblings, HLA-haploidentical bone marrow from family members, or HLA-matched unrelated donors. (medscape.com)
Bone marrow donors undergo a surgical procedure where the marrow containing the stem cells is collected from the pelvic bones. (blood.ca)
If there is no match in the family, we use donors from unrelated bone marrow registries, various international registries, like DKMS and DB, which is a US registry. (atheneum.ai)
Treatment with a new class of drugs, called senolytics, in donors improved the physical fitness of the recipients, a new study has shown. (medicaldaily.com)

Corneal Transplantation Corneal transplantations are done for several reasons: To reconstruct the cornea (eg, replacing a perforated cornea) To relieve intractable pain (eg, severe foreign body sensation due to recurrent. (msdmanuals.com)
Severe Immunodeficiencies: Bone Marrow Transplantation or Gene Therapy? (medscape.com)
Immune deficiencies occur infrequently in the immune system, but when they do, they are generally associated with severe manifestations owing to the lack of protections from life-threatening infections. (medscape.com)
They anticipate moving on to genes for drug resistance, factor IX, chronic granulomatous disease, the gamma chain for the T- cell receptor in severe combined immunodeficiency disease, and proteins that could inhibit replication of such viruses as HIV. (nih.gov)
Severe cases are known as favism because the consumption of broad beans can trigger a severe hemolytic episode in people who have a severe deficiency of this enzyme. (perfusiontheory.com)
Class II patients also have severe deficiency with periodic hemolysis. (perfusiontheory.com)

Because B lymphocytes were absent or low early after transplantation, the patient received immunoglobulins for 5 months (Fig. 1). (aacc.org)

Chronic blood loss is treated by identifying the source of bleeding, stopping the bleeding, and, if necessary, providing treatment for iron-deficiency anemia. (webmd.com)
In young women, iron deficiency anemia can result from heavy menstrual bleeding. (webmd.com)
With iron deficiency anemia, your doctor will probably recommend iron supplements that contain the ferrous form of iron. (webmd.com)
Note: Most patients/clients presenting with mild anemia [such as mild iron deficiency anemia] are likely to be medically cleared for dental hygiene procedures, because minor blood loss during procedures is likely to be well tolerated. (cdho.org)

[ 7 ] Other therapeutic options, such as gene therapy and bone marrow transplantation, for selected lipidoses may also result in improved prognosis. (medscape.com)
A long time ago, a cure was thought of as nothing less than changing the gene-getting rid of the deficiency so that subsequent generations would not be affected. (hemaware.org)

In patients with ADA deficiency, rigorous adherence to a schedule of ADAGEN ® (pegademase bovine) Injection administration can eliminate the toxic metabolites of ADA deficiency and result in improved immune function. (nih.gov)

Drugs, Essential General works QV 704 Economic aspects QV 736 Echocardiography, Four-Dimensional WG 141.5.E2 Echocardiography, Three-Dimensional WG 141.5.E2 Educational Technology LB 1028.3 Special topics, by subject Embolism, Paradoxical WG 540 Embolization, Therapeutic General works WH 310 Performed in interventional radiology WN 200 Used for the control of operative hemorrhage WO 500 Used for particular diseases, with the disease See also Hemostasis, Surgical WO 500, etc. (nih.gov)
A summary of the steps in the production of uric acid and genetic diseases associated with deficiencies of specific degradative enzymes are shown in Figure 22.15. (pharmacy180.com)
The growing list of resistant pathogens, nosocomial transmission and lack of an effective antifungal drug with a lower toxicity, are the potential problems associated with systemic fungal diseases. (ispub.com)

Inherited factor VII (FVII) deficiency is a rare autosomal recessive hemorrhagic disorder. (medscape.com)

For patients with leukemia or other cancers that cause bone marrow to function abnormally, chemotherapy, radiotherapy and specialized medications are tried first to stop the disease. (blood.ca)

Experiments in cells cultured from the patients suggested that ADA2 deficiency interferes with the body's balance of white blood cells called monocytes and macrophages. (nih.gov)
By working closely with patients, compounding pharmacists are able to provide correct dosages, strengths, and drug combinations for each individual situation. (ijpc.com)
In contrast to the natural history of combined immunodeficiency disease due to ADA deficiency, a trend toward diminished frequency of opportunistic infections and fewer complications of infections has occurred in patients receiving ADAGEN ® (pegademase bovine) Injection. (nih.gov)
Some of these same infections occurred in patients who received drugs to prevent rejection after kidney, heart or lung transplants. (healthworkscollective.com)
Early diagnosis and early implementation of bone marrow transplantation (BMT) are crucial to minimize neurodevelopmental complications and ensure productive adult life in patients with PNP deficiency. (medscape.com)
This is especially prevalent in PNP deficiency with neurologic symptoms, including mental retardation and muscle spasticity, reported in 67% of patients with PNP deficiency. (medscape.com)
Candida infects native cardiac valves in IV drug users and patients receiving parentral nutrition. (ispub.com)
For patients who do not improve with this drug, alternatives include dasatinib (Sprycel) and nilotinib (Tasigna). (empowher.com)
I wish we had a "magic bullet" drug effective enough to work for 100% of patients. (empowher.com)
Does Transplantation Induce Aging In Patients? (medicaldaily.com)
Class III patients have moderate deficiency with intermittent hemolysis associated with a stressor such as infection or drugs. (perfusiontheory.com)
Class IV patients have no deficiency or hemolysis. (perfusiontheory.com)

The donor cells will live in the recipient's bone marrow and make blood and immune system cells. (primaryimmune.org)

Recipients of grafts containing immune cells (particularly bone marrow, intestine, and liver) are at risk of graft-vs-host disease, in which the donor immune cells attack recipient tissue. (msdmanuals.com)
When a suitable bone marrow donor is unavailable or when bone marrow transplantation fails, non-selective replacement of the ADA enzyme has been provided by periodic irradiated red blood cell transfusions. (nih.gov)
She underwent bone marrow transplantation from a matched unrelated donor after receiving conditioning with fludarabine, treosulfan, and thiotepa, together with thymoglobulin, a rabbit antithymocyte globulin. (aacc.org)

For instance, early diagnosis and treatment is important to prevent potentially irreversible neurologic signs in vitamin B 12 and folate deficiency anemias. (cdho.org)

Immune deficiencies are generally classified depending on the arm of the immune system that is impaired. (medscape.com)
Immune system cells develop from special cells that live in the bone marrow called hematopoietic stem cells. (primaryimmune.org)

The researchers call the new syndrome Deficiency of ADA2, or DADA2. (nih.gov)
It was just 30 years ago in June 1981 when the first cases of what came to be known as Acquired Immune Deficiency Syndrome (AIDS) were published by the Centers for Disease Control (CDC) in its Morbidity and Mortality Weekly Report (MMWR. (healthworkscollective.com)

A 9-year-old girl was admitted to our hospital with juvenile metachromatic leukodystrophy (arylsulfatase A deficiency). (aacc.org)

Her laboratory research focuses on understanding how inflammation in the bone marrow environment can facilitate the outgrowth of clonally mutated hematopoietic stem cells (HSCs) and contribute to the early development of myeloid leukemias. (childrens.com)
For a bone marrow donation, the collection of the stem cells is taken from the iliac crest which sits at the back of the pelvic bone. (blood.ca)
The blood is passed through a machine that separates out the stem cells used in transplantation. (blood.ca)
A needle is inserted to draw out the rich and immature blood stem cells which are processed, frozen and stored for transplantation to a matched patient. (blood.ca)

This case represents a rare complication of antituberculous vaccination, that is a progressive, disseminated BCG infection in a patient with deficiency of IFN-γ receptor. (cdc.gov)
By 1987 the first of the antiviral drugs zidovudine (or AZT) to actually treat the underlying HIV infection was approved by the FDA after studies began at Burroughs Welcome by David Barry, MD and others and at the National Cancer Institute by Samuel Broder, MD and others. (healthworkscollective.com)
The Food and Drug Administration (FDA) has licensed 2 vaccines for the prevention of monkeypox infection: ACAM2000 (smallpox [vaccinia] vaccine, live) and Jynneos (smallpox and monkeypox vaccine, live, nonreplicating). (clinicaladvisor.com)
Hair loss, nausea, and vulnerability to infection are well-known side effects of this class of drugs. (empowher.com)
The hemolysis usually has a trigger such as certain foods, drugs, stressors or infection and older red cells are affected most because of their fragility. (perfusiontheory.com)

Furthermore, biomarkers approved by the U.S. Food and Drug Administration for screening in adult blood were detected in UCB and represent high-priority targets for immediate validation. (nih.gov)
He specializes in Stem Cell transplantation and management of blood and cancer disorder and has undertaken international fellowships at the Imperial College London Healthcare Trust in the UK. (atheneum.ai)

If you have ever had a patient on cardiopulmonary bypass develop dark, wine colored urine or an ECMO patient with persistent hemolysis without a readily recognizable cause, a G6PD deficiency may be the reason. (perfusiontheory.com)

Indications for liver transplantation include Cirrhosis (70% of transplantations. (msdmanuals.com)
Some research is ongoing in transplantation of non-human organs, such as heart, kidney and liver transplantation. (msdmanuals.com)
Drs. Thomas Randall (retired), Terry Waters and Jacqueline Trudeau are anesthesiologists with a special interest in liver transplantation. (liver.ca)

NIAID also supports the development of antifungal drugs and other therapies to curb infections and improve outcomes for people with CGD. (nih.gov)

Objective evidence of a platelet storage pool deficiency (e.g., an abnormal secondary aggregation response or absent platelet dense bodies) or of a lysosomal fusion abnormality (e.g., giant cytoplasmic granules in leucocytes) will not be required. (nih.gov)

In each disorder, a deficiency of a lysosomal hydrolase is inherited, which leads to lysosomal accumulation of the enzyme's specific sphingolipid substrate. (medscape.com)

PNP deficiency causes profound T lymphopenia and variable numbers of B and NK cells. (medscape.com)

The webinar event will feature Sonata Jodele, MD, Research Professor in the Division of Bone Marrow Transplantation and Immune Deficiency at the University of Cincinnati Department of Pediatrics and Professor Rob Wynn of Royal Manchester Children's Hospital, part of Manchester University NHS Foundation Trust. (woodtv.com)

whereas in PNP deficiency, the metabolites are especially toxic to T-lineage cells, resulting in profound T-cell deficiency and variable degree of B-cell dysfunction. (medscape.com)

An artificial form of the protein or bone marrow transplantations might also help. (nih.gov)

It is caused by a deficiency of N-ACETYLGALACTOSAMINE-4-SULFATASE (arylsulfatase B). (curehunter.com)

Deficiency or defects in Apo E are causes of HYPERLIPOPROTEINEMIA TYPE III. (curehunter.com)

Anatomy29

Organisms14

Diseases63

Chemicals and Drugs38

Analytical, Diagnostic and Therapeutic Techniques and Equipment73

Psychiatry and Psychology2

Phenomena and Processes39

Disciplines and Occupations2

Anthropology, Education, Sociology and Social Phenomena2

Information Science3

Named Groups3

Health Care14

Allogeneic bone marrow tra1

Transplant10

Umbilical cord3

Hematopoietic stem8

Disorders11

Acquired immunodeficiency2

IMMUNOLOGY1

Purine nucleoside3

Organ3

SCID9

Glucose-6-phosphate d1

Autologous2

Hemolytic anemia1

Oncology1

Abnormalities2

HSCT1

Adenosine deaminase1

Lymphoma2

Immunosuppressive2

Enzyme deficiency1

Clinical4

Peripheral4

Neutropenia2

Donors4

Severe6

Lymphocytes1

Iron defici4

Gene2

Toxic metabolites1

Diseases3

Autosomal recessive1

Leukemia1

Patients12

Recipient's1

Donor3

Neurologic1

Immune system2

Syndrome2

Metachromatic leukodystrophy1

Stem cells4

Infection5

Blood2

G6PD1

Liver3

Antifungal1

Abnormal1

Lysosomal1

Lymphopenia1

Pediatrics1

Dysfunction1

Protein1

Arylsulfatase1

Defects1