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FDA Grants Rare Pediatric Disease Designation to LSTA1 in Osteosarcoma

The investigational drug LSTA1 is being explored for the treatment of osteosarcoma, which starts in the bones and accounts for 2% of childhood cancers.

United statesDrug administrationAmerican cancer societyRare pediatric designationRare pediatric disease designationRare pediatric disease priority review voucherRare pediatric drug designationFast track

Andelyn Biosciences, Inc : Andelyn Biosciences and Armatus Bio Partner to Manufacture Suspension-Based AAV Gene Therapy for Rare Neurological Disease Charcot-Marie-Tooth Type 1A (CMT1A)

Andelyn Biosciences, Inc : Andelyn Biosciences and Armatus Bio Partner to Manufacture Suspension-Based AAV Gene Therapy for Rare Neurological Disease Charcot-Marie-Tooth Type 1A (CMT1A)
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United statesAndelyn biosciencesMatt niloffArmatus bioBrian priceAndelyn biosciences incPrnewswire andelyn biosciences incContract developmentManufacturing organizationCharcot marie tooth typeChief commercial officerChief technical officerOrphan drug designationRare pediatric drug designationFacioscapulohumeral muscular dystrophy

HUIADGENE ANNOUNCES RARE PEDIATRIC DRUG DESIGNATION GRANTED TO HG302, A NOVEL CRISPR DNA-EDITING THERAPY, FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

HUIADGENE ANNOUNCES RARE PEDIATRIC DRUG DESIGNATION GRANTED TO HG302, A NOVEL CRISPR DNA-EDITING THERAPY, FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
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Calvin lukNgene therapeuticsRare pediatric drug designationLicensing application

IPS HEART Receives U S FDA Rare Pediatric Drug Designation for ISX9-CPC Stem Cell Therapy for Treatment of Cardiomyopathy Associated with Danon disease

IPS HEART Receives U S FDA Rare Pediatric Drug Designation for ISX9-CPC Stem Cell Therapy for Treatment of Cardiomyopathy Associated with Danon disease
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Stephanie carringtonRauf ashrafDrug administrationRare pediatric drug designationOrphan drug designationChief executive officerDuchenne cardiomyopathy

IPS HEART Receives U S FDA Rare Pediatric Drug Designation for ISX9-CPC Stem Cell Therapy for Treat

Marks third Rare Pediatric Drug Designation granted by FDA for pipeline candidateHOUSTON (BUSINESS WIRE) IPS HEART, a privately held cell therapy company advancing its stem cell platform to develop new skeletal muscle and cardiac muscle generation treatments for Duchenne muscular dystrophy (a rare disease) and hea.

Rauf ashrafStephanie carringtonDrug administrationRare pediatric drug designationOrphan drug designationChief executive officerDuchenne cardiomyopathy