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Peer-Reviewed Publication of Positive Preclinical Data of SiSaf s SIS-ADO2 siRNA Program to Treat Rare Genetic Bone Disorder Osteopetrosis

SiSaf Ltd, an RNA therapeutics company, is pleased to announce the publication in the peer-reviewed journal Molecular Therapy Nucleic Acids(1) of positive in vivo data demonstrating the safety and efficacy of its siRNA therapy for Autosomal Dominant Osteopetrosis 2 (ADO2). The data reported in the paper demonstrate .

United statesAnna maria tetiSuzanne saffie siebertAntonio mauriziSisaf ltdMineral researchAmerican society of boneCell press journal molecular therapy nucleic acidsCell press journal molecular therapy nucleicMolecular therapy nucleic acidsAutosomal dominant osteopetrosisAmerican societyProfessor anna mariaProfessor tetiOrphan drug designationPediatric disease designation

Peer-Reviewed Publication of Positive Preclinical Data of SiSaf s SIS-ADO2 siRNA Program to Treat Rare Genetic Bone Disorder Osteopetrosis

Peer-Reviewed Publication of Positive Preclinical Data of SiSaf s SIS-ADO2 siRNA Program to Treat Rare Genetic Bone Disorder Osteopetrosis
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United kingdomUnited statesSue charlesAnna maria tetiAntonio mauriziSuzanne saffie siebertMineral researchAmerican society of boneSisaf ltdReviewed publicationPositive preclinical dataTreat rare genetic bone disorderSaf ltdMolecular therapy nucleicAutosomal dominant osteopetrosisAmerican society

Kidney disease patients needed for national trial

Kidney disease patients needed for national trial
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David johnsonAndrew mallettUq institute for molecular bioscienceAustralasian kidney trials networkTownsville university hospitalProfessor andrew mallettMolecular bioscienceProfessor david johnsonAutosomal dominant

Pfeiffer Syndrome - Causes, Symptoms, Diagnosis, Treatment & Prevention

Pfeiffer syndrome is a rare genetic disorder with fused skull bones and fused feet and hands. Multidisciplinary care helps to manage the syndrome.

United statesSmithas duttRudolf arthur pfeifferBurle aguiar mj pfeifferResearch in autism spectrum disorderGroup on craniosynostosisUltrasonography helpsDetect pfeifferMachador aColettar dPatients withWorking groupPfeiffer syndromeMolecular genetics diagnosticAutism spectrumGenetic counseling

UChicago Medicine receives Center of Excellence designation from Polycystic Kidney Disease Foundation

UChicago Medicine was named a Center of Excellence by the Polycystic Kidney Disease (PKD) Foundation for its work caring for patients with Autosomal Dominant PKD, a life-threatening genetic disease that causes kidney failure.

Chris rusconiDarlene chapmanCenters of excellenceUniversity of chicago medicineChicago medicinePolycystic kidney diseaseAutosomal dominantSouth sideInterim chief executive officerChief research officer