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Enzyme replacement therapy

Ultragenyx’s Gene Therapy Ameliorates Pediatric Neurodegenerative Disorder
Drug Discovery

Ultragenyx’s Gene Therapy Ameliorates Pediatric Neurodegenerative Disorder

Gene Therapy for ADA-SCID Still Benefitting Patients after Two to Three Years
Genome Editing

Gene Therapy for ADA-SCID Still Benefitting Patients after Two to Three Years

Sangamo Shares Plunge after Interim Results for MPS Candidates Fall Short of Clinical Benefit
Featured

Sangamo Shares Plunge after Interim Results for MPS Candidates Fall Short of Clinical Benefit

Gene Therapy for Rare Diseases
Insights

Gene Therapy for Rare Diseases

FDA Approves Ultragenyx Enzyme Replacement Therapy for MPS VII
Industry News

FDA Approves Ultragenyx Enzyme Replacement Therapy for MPS VII

Rubius Therapeutics Closes $120M Financing, Plans Trials Next Year
Cancer

Rubius Therapeutics Closes $120M Financing, Plans Trials Next Year

mRNA: Addressing the Blind Spot of Enzyme Replacement Therapy
Insights

mRNA: Addressing the Blind Spot of Enzyme Replacement Therapy

Protalix Sells Share of ELELYSO Collaboration, Equity Stake to Pfizer
Drug Discovery

Protalix Sells Share of ELELYSO Collaboration, Equity Stake to Pfizer

AbbVie, Shire Call Off $51.7B Merger
Industry News

AbbVie, Shire Call Off $51.7B Merger

ArmaGen Licenses AGT-182 Rights to Shire for Up to $225M
News

ArmaGen Licenses AGT-182 Rights to Shire for Up to $225M

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