Latest Breaking News On - Paediatric inherited metabolic disease - Page 1 : comparemela.com

NICE recommends first treatment for infants with Wolman Disease

NICE recommends first treatment for infants with Wolman Disease
europeanpharmaceuticalreview.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from europeanpharmaceuticalreview.com Daily Mail and Mail on Sunday newspapers.

United kingdomBob stevensSimon jonesFoundation trustEuropean medicines agencyDrug administrationNational institute for healthManchester universityNational instituteCare excellenceAstrazeneca rare diseaseWolman diseaseGroup chief executiveClinical research facilityRoyal manchester childrenProfessor simon jones

NICE endorses only approved treatment for ultra-rare Wolman disease for children diagnosed under the age of two: Interview Opportunity

NICE endorses only approved treatment for ultra-rare Wolman disease for children diagnosed under the age of two: Interview Opportunity

United kingdomCity ofUnited statesSean richardsonBob stevensAnna clarkSimon jonesNational health serviceUniversity dental hospital of manchesterManchester universityDepartment of healthEuropean medicines agencySaint mary managed clinical serviceEuropean unionAlexion pharmaceuticals incFoundation trust

NICE recommends first treatment for infants for Wolman Disease

NICE recommends first treatment for infants for Wolman Disease
europeanpharmaceuticalreview.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from europeanpharmaceuticalreview.com Daily Mail and Mail on Sunday newspapers.

United kingdomBob stevensSimon jonesManchester universityNational institute for healthFoundation trustDrug administrationEuropean medicines agencyNational instituteCare excellenceAstrazeneca rare diseaseWolman diseaseGroup chief executiveClinical research facilityRoyal manchester childrenProfessor simon jones

Revolutionary Gene Therapy Trial Begins for Hunter Syndrome

Revolutionary Gene Therapy Trial Begins for Hunter Syndrome
miragenews.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from miragenews.com Daily Mail and Mail on Sunday newspapers.

United kingdomSimon jonesBob stevensRob wynnResearch ethics committeeManchester universityFoundation trustHealthcare products regulatory agencyUniversity of manchesterHealth research authorityRoyal manchester childrenManchester centreGenomic medicineSaint maryBrian biggerGene therapy

Novel Gene Therapy for Hunter Syndrome

The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder.

United kingdomSimon jonesBob stevensRob wynnUniversity of manchesterManchester universityFoundation trustTechnology networkResearch ethics committeeHealth research authorityHealthcare products regulatory agencyRoyal manchester childrenManchester centreGenomic medicineSaint maryBrian bigger