While gene therapy offers the promise of addressing and treating many genetic disorders, creating a better quality of life for many people, there are also ethical issues associated with…
Armené and Michael Kapamajian live in LA. Their son Sasoun was diagnosed with severe combined immunodeficiency (SCID). This means that his immune system is compromised and he is extremely vulnerable…
According to a story from gurufocus.com, the biopharmaceutical company ADMA Biologics, Inc., has earned the 2020 BioNJ Innovator Award. This award was given as acknowledgement of the company's successful development…
Mustang Bio, a biopharmaceutical company, has been working with St. Jude Children's Research Hospital to develop MB-107, a lentiviral gene therapy to treat X-linked severe combined immunodeficiency. Mustang Bio…
The Bettuchi's Amanda Bettuchi and her husband have 3 children. Their middle child, Shane, was born with severe combined immunodeficiency (SCID). Essentially, this means he was born with practically no…
According to a story from Express Digest, four year old Khloe Land of Oregon stepped up to the plate to help her brother get a critical treatment that could very…
According to a press release from American biotechnology company Fortress Biotech, the American Food and Drug Administration (FDA) recently awarded Regenerative Medicine Advanced Therapy designation to the experimental gene therapy…
ABC News and Good Morning America brightened up our morning by featuring a story of 1-year-old Alle Jilg, whose life was saved by total stranger's bone marrow donation. Alle is…
According to a story from Australasian Science Magazine, the dawn of the gene therapy "revolution" is upon us. While there are only a handful of gene therapies that have actually…
SCID Severe combined immunodeficiency (SCID) is a rare disease which causes patients to have little to no immune response. With practically no immune system, SCID patients are very susceptible to…
Researchers at the University of Colorado Anschutz Medical Campus have uncovered a never-before discovered immunodeficiency disease. How It Came About It started with a newborn boy who had a myriad…
We just read exciting news for the SCID community from Stanford researchers. The original study was published in Nature Communications, but a summary appeared in Medical Xpress. The researchers have…
ADA-SCID ADA-SCID is a form of Severe Combined Immunodeficiency (SCID) which is caused by Adenosine Deaminase Deficiency. A lack of the ADA gene in the body results in impaired development of the…
According to a story from news-medical.net, a team of researchers from UCLA have successfully developed an approach that allows for the reversal of the mutation that is responsible for the…
When you're told your child has a rare, life-threatening condition you exhaust every resource trying to find answers. But the rarer the condition, the harder those answers are to find…
A frequent impediment to rare disease research is cost. It's unfortunate, but it's the reality of the world we live in. That means every fundraising campaign and every dollar raised…
Maurice Elias, who was born with severe combined immunodeficiency, has met up with the doctor who saved his life almost fifty years ago. For more information, you can read the…
According to a report by BioSpace, a new treatment for SCID could be around the corner. Mustang Bio, Inc. (Mustang), and St. Jude Children’s Research Hospital recently announced a partnership…
Researchers are exploring a new diagnostic method that could be applied to newborn screening for certain inherited diseases and monitoring of HIV patients. For more detailed information, you can click…
A gene therapy treatment is being used to treat X-linked severe combined immunodeficiency (X-SCID). Mustang Bio has licensed the treatment from St. Jude Children's Research Hospital. X-SCID is also known…
What could make a family in Lebanon travel all the way to UCLA? The answer is a unique stem cell gene therapy designed to combat SCID. The El Kerdi family…
What could make a family in Lebanon travel all the way to UCLA? The answer is a unique stem cell gene therapy designed to combat SCID. The El Kerdi family…
A primary immunodeficiency report, published in the journal Clinical and Experimental Immunology, examines medical care for primary immunodeficiency patients in the UK. The publication of the report coincides with the…
Researchers from St. Jude Children's Hospital might have just developed a new gene therapy that can build a healthy, functioning immune system for patients suffering from severe combined immunodeficiency, SCID,…
'Tis the season for gene therapy approvals. Reported by Bloomberg, the U.K. just approved its most expensive medication to date. The treatment is for severe combine immune deficiency, or more…
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