Severe Combined Immunodeficiency News and Research

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Novel gene therapy can improve symptoms of Bubble Boy disease in young adults

Adolescents and young adults with a severe inherited immunodeficiency disorder improved following treatment with novel gene therapy developed at St. Jude Children's Research Hospital and at the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health. The results of this study appear today in the journal Science Translational Medicine.

21 Apr 2016

Gene therapy can restore immune systems of children and young adults with SCID-X1

Gene therapy can safely rebuild the immune systems of older children and young adults with X-linked severe combined immunodeficiency (SCID-X1), a rare inherited disorder that primarily affects males, scientists from the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, have found.

7 Dec 2015

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

New research adds to a growing body of evidence that gene therapy, an experimental technique that involves correcting or replacing a person's mutated or malfunctioning genes, may improve health outcomes for patients with inherited bleeding and immune disorders as well as some forms of blood cancer.

7 Dec 2015

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Prolong Pharmaceuticals, LLC, a biopharmaceutical company dedicated to developing products for the treatment of anemias, cancers and their debilitating comorbidities, announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation for its flagship product SANGUINATE for the treatment of Sickle Cell Disease (SCD).

8 Apr 2015

Study: New gene therapy safe, effective for patients with hemophilia B

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding.

12 Mar 2015

Finding could lead to more effective, less invasive treatment for 'bubble boy' disease

For infants with severe combined immunodeficiency (SCID), something as simple as a common cold or ear infection can be fatal. Born with an incomplete immune system, kids who have SCID--also known as "bubble boy" or "bubble baby" disease--can't fight off even the mildest of germs.

12 Mar 2015

UCSD professor wins 2015 Japan Prize

Theodore Friedmann, MD, professor in the Department of Pediatrics at University of California, San Diego School of Medicine was named today one of three recipients of the 2015 Japan Prize, a prestigious international award honoring laureates whose "original and outstanding achievements in science and technology have advanced the frontiers of knowledge and served the cause of peace and prosperity for mankind.

29 Jan 2015

Gut microbe in patients may tip doctors about SCID

Many people recognize "the bubble boy" as an unusual character from a "Seinfeld" episode or a John Travolta movie.

30 Sep 2014

Newborn screening for SCID holds promise that affected children can lead healthy lives

Using population-based screening outcomes of approximately 3 million infants, a team of scientists across 14 states, including four researchers at the University of Massachusetts Medical School, have shown that newborn screening for severe combined immunodeficiency (SCID) can be successfully implemented across public health newborn screening programs.

21 Aug 2014

Study examines national impact of newborn screening test for SCID

Severe combined immunodeficiency (SCID), a potentially life-threatening, but treatable, disorder affecting infants, is twice as common as previously believed, according to a new study that is the first to examine the national impact of this newborn screening test.

20 Aug 2014

Newborn screening indicates that incidence of SCID is higher than previously believed

Newborn screening performed in numerous states indicates that the incidence of the potentially life-threatening disorder, severe combined immunodeficiency, is higher than previously believed, at 1 in 58,000 births, although there is a high rate of survival, according to a study in the August 20 issue of JAMA.

20 Aug 2014

Blood-forming stem cell transplantation benefits infants with severe combined immunodeficiency

Babies who are born with severe combined immunodeficiency (SCID) can be successfully treated with a transplant of blood-forming stem cells, according to experts led by Memorial Sloan Kettering's Richard J. O'Reilly, MD, a world-renowned pioneer in the development of transplant protocols.

31 Jul 2014

Two pediatricians win March of Dimes/Col. Harland D. Sanders Lifetime Achievement Award in Genetics

Two pediatricians whose research led to the need for early identification of and treatment of severe combined immunodeficiency (SCID) and its inclusion in newborn screening have received the March of Dimes/Col. Harland D. Sanders Lifetime Achievement Award in Genetics.

29 Mar 2014

New form of gene therapy shows promising outcomes for "bubble boy" disease

Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as "bubble boy" disease, who were treated as part of an international clinical study of a new form of gene therapy. The mechanism used to deliver the gene therapy is designed to prevent the serious complication of leukemia that arose a decade ago in a similar trial in Europe, when one-quarter of boys treated developed the blood cancer.

9 Dec 2013

Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

A series of advancements in genetically engineered cell therapies demonstrate early efficacy and safety in patients with blood disorders for whom standard treatments have been unsuccessful, according to data showcased today during the 55th American Society of Hematology (ASH) Annual Meeting and Exposition in New Orleans.

9 Dec 2013

EMA expands administration options for Hizentra

CSL Behring UK Ltd announced today that the European Medicines Agency (EMA) has expanded the administration options for Hizentra®▼, human normal immunoglobulin, SCIg, 20% liquid, to include dosing once every two weeks (fortnightly).

5 Dec 2013

Scientists describe interplay of four molecules that trigger, govern antibody production in immune cells

Australian scientists have described an exquisitely balanced interplay of four molecules that trigger and govern antibody production in immune cells. As well as being an important basic science discovery, it helps explain why people with mutations in any one of the associated genes cannot fight infection effectively, and develop rare and crippling immunodeficiency disorders.

18 Nov 2013

UCSF to receive grant to study gene sequencing aimed at detecting disorders in newborns

UC San Francisco will receive $4.5 million over the next five years for a pilot project to assess whether large-scale gene sequencing aimed at detecting disorders and conditions can and should become a routine part of newborn testing.

6 Sep 2013

Researchers expand hematopoietic stem cells for bone marrow transplantation

More than 50,000 stem cell transplants are performed each year worldwide. A research team led by Weill Cornell Medical College investigators may have solved a major issue of expanding adult hematopoietic stem cells (HSCs) outside the human body for clinical use in bone marrow transplantation -- a critical step towards producing a large supply of blood stem cells needed to restore a healthy blood system.

25 Mar 2013

Early lymphoid progenitors thrive in an osteoblastic niche: Study

The Children's Medical Center Research Institute at UT Southwestern has deepened the understanding of the environment within bone marrow that nurtures stem cells, this time identifying the biological setting for specialized blood-forming cells that produce the infection-fighting white blood cells known as T cells and B cells.

25 Feb 2013