Patients with Newly Diagnosed Fanconi Anemia Should Avoid alloHSCT

A study in the American Journal of Hematology recently presented some troubling findings on the relationship between Fanconi anemia and allogeneic hematopoietic stem cell transplantation (alloHSCT). Patients who participated in alloHSCT before achieving…

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Investigative Gene Therapy for Fanconi Anemia Receives PRIME Designation

A Fanconi Anemia (FA) investigative gene therapy now has PRIority MEdicines (PRIME) designation from the EMA. This designation helps to accelerate the development of therapies for patient populations who have…

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Experimental Gene Therapy for Fanconi Anemia Earns Fast Track and Regenerative Medicine Advanced Therapy Designation

According to a story from BioPortfolio, the gene therapy company Rocket Pharmaceuticals has recently announced that the company's investigational gene therapy RP-L102 has earned both Fast Track and Regenerative Medicine…

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Gene Therapy for Fanconi Anemia Gains IND Clearance From the FDA
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Gene Therapy for Fanconi Anemia Gains IND Clearance From the FDA

According to a story from BioSpace, the gene therapy company Rocket Pharma recently announced that its investigational product candidate RP-L102 recently saw its Investigational New Drug (IND) application gain approval…

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New Aldehyde Exposure Test Could Help Patients with Fanconi Anemia and Similar Diseases
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New Aldehyde Exposure Test Could Help Patients with Fanconi Anemia and Similar Diseases

According to a story from Check Orphan, a group of scientists from Stanford have begun the process of developing a new test that could help patients with rare Fanconi anemia…

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