Severe Combined Immunodeficiency News and Research

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New gene therapy cures babies with fatal 'Bubble Boy' disease

A new gene therapy created by US researchers appears to have cured eight infant boys born with a rare genetic disorder referred to as “Bubble Boy” disease.

18 Apr 2019

Antibody-based treatment could improve cure rate for blood and immune disorders

An antibody-based treatment can gently and effectively eliminate diseased blood-forming stem cells in the bone marrow to prepare for the transplantation of healthy stem cells, according to a study in mice by researchers at the Stanford University School of Medicine.

12 Feb 2019

Leadiant Biosciences announces FDA approval of Revcovi for treating pediatric, adult ADA-SCID patients

Leadiant Biosciences, Inc. today announced that the Food and Drug Administration has granted approval to Revcovi (elapegademase-lvlr) injection in the U.S. Revcovi is a new enzyme replacement therapy for the treatment of adenosine deaminase severe combined immune deficiency in pediatric and adult patients.

7 Oct 2018

Using novel gene therapy to cure infants born with severe combined immunodeficiency

Out of every 60,000 births, a baby arrives to face the world without a fully functioning immune system leaving them unequipped to fight even the most common infections.

29 Aug 2018

Smart Cells launches social campaign to support Rare Disease Day 2018

There are more than 6000 known rare diseases and over 30 million people living daily with them. 28th February marks Rare Disease Day 2018, a date that highlights awareness around these conditions and the need for more research into them.

From Smart Cells International Ltd 23 Feb 2018

A guide to Severe Combined Immunodeficiency Disease, review released

A new review published today in the Canadian Medical Association Journal provides guidance on Severe Combined Immunodeficiency Disease (SCID), a deadly and rare disease that is potentially curable if detected early.

18 Dec 2017

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

A new review provides guidance on a deadly, but rare, disease that is potentially curable if identified early. Severe combined immunodeficiency disease (SCID), known as the "bubble boy disease" in the 1970s, is treatable with a stem cell transplant, gene therapy and other treatments if identified at birth or soon after.

18 Dec 2017

St. Jude gene therapy offers hope for infants with 'bubble boy' disease

Early evidence suggests that gene therapy developed at St. Jude Children's Research Hospital will lead to broad protection for infants with the devastating immune disorder X-linked severe combined immunodeficiency disorder.

11 Dec 2017

Orchard Therapeutics opens new facility for technical operations in San Francisco Bay area

Orchard Therapeutics, a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies, today announced the opening of a second facility for technical operations in the San Francisco Bay Area.

1 Dec 2017

Study to determine lowest dose of chemotherapy needed for babies with SCID

Michael Pulsipher, MD, of the Children's Center for Cancer and Blood Diseases at Children's Hospital Los Angeles, along with co-principal investigator, Sung-Yun Pai, MD, of Boston Children's Hospital, has been awarded nearly $9 million from the National Institute of Allergy and Infectious Diseases of the NIH to study a new treatment approach for babies born with severe combined immunodeficiency (SCID) which prevents the immune system from functioning normally.

6 Oct 2017

Moms of children with rare genetic illness push for wider newborn screening

Kerri De Nies received the news this spring from her son's pediatrician: Her chubby-cheeked toddler had a rare brain disorder.

5 Oct 2017

IQWiG assesses benefit of newborn screening for severe combined immunodeficiency

The German Institute for Quality and Efficiency in Health Care assessed the benefit of newborn screening for severe combined immunodeficiency (SCID).

30 Jan 2017

Researchers develop first non-human primate X-SCID models using genome editing techniques

Researchers affiliated with the Kawasaki INnovation Gateway at SKYFRONT, have successfully generated the first ever non-human primate X-SCID models by using two genome editing techniques. The findings were published in Cell Stem Cell, July 2016.

3 Jan 2017

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients.

12 Oct 2016

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.