Gene Therapy Promising for Cerebral Adrenoleukodystrophy but Carries Risk of Blood Cancer
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Gene Therapy Promising for Cerebral Adrenoleukodystrophy but Carries Risk of Blood Cancer

The use of the gene therapy elivaldogene autotemcel (Skysona), has been approved due to its success in the treatment of patients diagnosed with the neurological disease cerebral adrenoleukodystrophy (CALD). The…

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ICYMI: Neuraxpharm and Minoryx’s Special Information Film Raises Leukodystrophy Awareness
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ICYMI: Neuraxpharm and Minoryx’s Special Information Film Raises Leukodystrophy Awareness

Rare Disease Day may be long past in February, but the fight for rare disease awareness continues. Earlier this year, specialty pharmaceutical company Neuraxpharm Group ("Neuraxpharm") and biotechnology company Minoryx…

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Gene Therapy for Cerebral Adrenoleukodystrophy Earns FDA Accelerated Approval
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Gene Therapy for Cerebral Adrenoleukodystrophy Earns FDA Accelerated Approval

According to a press release, the gene therapy company bluebird bio, inc. has recently announced that its gene therapy elivaldogene autotemcel (marketed as SKYSONA) has received Accelerated Approval status from…

Continue Reading Gene Therapy for Cerebral Adrenoleukodystrophy Earns FDA Accelerated Approval
New Trial Data Bodes Well for Experimental Cerebral Adrenoleukodystrophy Gene Therapy
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New Trial Data Bodes Well for Experimental Cerebral Adrenoleukodystrophy Gene Therapy

According to a story from pmlive.com, the gene therapy company Bluebird Bio's latest recently released results from the company's phase 2/3 clinical trial demonstrates further encouraging data for its Lenti-D…

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Results From a Study of Gene Therapy for Cerebral Adrenoleukodystrophy Appear Positive
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Results From a Study of Gene Therapy for Cerebral Adrenoleukodystrophy Appear Positive

According to a story from PharmaTimes, the gene therapy company Bluebird bio has released updated results from a clinical study of its Lenti-D gene therapy as a treatment for patients…

Continue Reading Results From a Study of Gene Therapy for Cerebral Adrenoleukodystrophy Appear Positive