Possible XSCID Treatment MB-207 Granted Orphan Drug Designation
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Possible XSCID Treatment MB-207 Granted Orphan Drug Designation

  Just last week, biopharmaceutical company Mustang Bio announced Orphan Drug designation for its therapeutic candidate, MB-207. This unique lentiviral gene therapy is designed for previously treated patients with X-linked…

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MB-107 Granted Rare Pediatric Disease Designation for X-Linked Severe Combined Immunodeficiency
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MB-107 Granted Rare Pediatric Disease Designation for X-Linked Severe Combined Immunodeficiency

  This past week, biopharmaceutical company Mustang Bio announced that their gene therapy candidate MB-107 received Rare Pediatric Disease Designation. The gene therapy candidate is designed to treat X-linked severe…

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Severe Combined Immunodeficiency: After Quarantine With Their Baby, This Family Was Prepared for Coronavirus
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Severe Combined Immunodeficiency: After Quarantine With Their Baby, This Family Was Prepared for Coronavirus

  Michael and Armené Kapamajian were at home in Los Angeles settling in with their one week old baby, Sasoun, when their doctor called and asked them to come in…

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Gene Therapy: The New Frontier for Cancer

  Gene therapy is the treatment of disease by way of the transfer of genetic material into cells. According to a recent article in Labiotech, an EU publication, gene therapy…

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Five Month Old Patient in the Spotlight for Primary Immunodeficiency Walk
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Five Month Old Patient in the Spotlight for Primary Immunodeficiency Walk

According to a story from the Warwick Beacon, the first ever primary immunodeficiency (PI) awareness and fundraising walk in Rhode Island's history was held recently at Warwick City Park. The…

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Family is Searching for an Eligible Bone Marrow Donor for Their Infant with Severe Combined Immunodeficiency

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…

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Eight Children with Severe Combined Immunodeficiency Cured with Gene Therapy

Severe Combined Immunodeficiency Severe Combined Immunodeficiency (SCID) is a rare disease which causes patients to practically have no immune system at all. That means that for these patients, even a…

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Analysis of Phase 3 Study Shows Inolimomab has Long-term Clinical Benefit for Graft-Versus-Host-Disease

About Graft-Versus-Host-Disease (GvHD) Graft-Versus-Host-Disease (GvHD) occurs when a patient's body rejects cells which have been transplanted. For instance, it may develop following a stem cell transplant or a bone marrow transplant. The…

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Lebanese Family Travels Across the World to Find Treatment for Child with SCID
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Lebanese Family Travels Across the World to Find Treatment for Child with SCID

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…

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Researchers Use Gene Therapy To Build Immune Systems For Patients With Bubble-Boy Disease
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Researchers Use Gene Therapy To Build Immune Systems For Patients With Bubble-Boy Disease

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,…

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