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Drug Prototype Promising in Treatment of Pulmonary Disease

Proof-of-concept study supported potential of drug-like small molecule to promote lung tissue repair.

United statesSan diegoBaylor college of medicineMichaelj bollongDharanik narendraDepartment of chemistry at scripps researchCalibr skaggs institute for innovative medicinesProceedings of the national academy sciencesScripps researchNational academyCalibr skaggs instituteInnovative medicinesMedscape medicalBaylor collegeIdiopathic pulmonary fibrosisDiopathic pulmonary fibrosis ipf

Better Outcomes With Early HETs in Pediatric-Onset MS

Early initiation of HET in pediatric-onset MS was linked to slower progression to disability compared to treatment with low-efficacy therapy or no treatment.

Sifat sharmanE ann yehDepartment of medicineUniversity of melbourneEuropean medicines agencyDemyelinating disorders programUniversity of torontoDrug administrationLancet childItalian multiple sclerosisRelated disorders registerExpanded disability status scalePediatric multiple sclerosisMultiple sclerosis msS multiple sclerosisBiologic therapy

Multiple Myeloma: What Treatments Work Best?

The longest overall survival of nearly 8-10 years was with autologous stem cell transplantation.

Niels abildgaardOdense university hospitalTakeda pharmaceuticals internationalEuropean journalMultiple myelomaPlasma cell myelomaTransplantation of stem cellsRansplantation of hematopoietic stem cellsHematopoietic stem cell transplantStem cell transplantationStem cell therapyTem cell haematopoietic stem transplantSct hematopoietic stem cell transplantation hsct haematopoietic

Vertex Pauses Islet Cell Study After Patient Deaths

The deaths are unrelated to the investigational allogeneic stem cell–derived product VX-880, the company says.

New yorkUnited statesEuropean association for the study of diabetesVertex pharmaceuticals incAmerican diabetes associationNew york timesVertex pharmaceuticalsMedscape medical newsDiabetes associationScientific sessionsTypei diabetesMassachusetts general hospitalDiabetes mellitus type 1Iabetes mellitus type iType 1 diabetesYpe 1 dm

EU Conditional Approval: Gene Editor to Treat Blood Diseases

The single-dose therapy involves treating and editing patient stem cells ex vivo, then reinfusing them.

Vertex pharmaceuticals irelandEuropean medicines agencyDrug administrationMore researchSickle cell diseaseIckle cell anemia sickle anaemiaClustered regularly interspaced short palindromic repeatsCrispr cas9Gene editingGenetic editingRare diseasesTransplantation of stem cellsRansplantation of hematopoietic stem cellsHematopoietic stem cell transplantStem cell transplantationStem cell therapy

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