TTR amyloidosis

The effort will provide individuals with genetic testing to determine if they have the disorder, and when necessary,  with genetic testing counselling.

The probes let researchers analyze oligomeric aggregates linked to forms of amyloidosis and could aid early diagnosis or monitoring response to therapy.

Through this Alnylam-funded program, Invitae will perform free testing for US patients suspected to have hereditary ATTR amyloidosis or a family history of it. 

The firm also said it aims to soon begin human studies for its hepatic porphyria treatment ALN-AS1 and to select a development candidate for its program in primary hyperoxaluria type 1.

All of the adverse events were deemed mild and are not expected to affect the drug's future clinical development.

 

NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals announced this week that it has completed enrollment in a Phase II trial of its subcutaneously administered TTR-mediated amyloidosis (ATTR) drug revusiran, formerly known as ALN-TTRsc.

NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week released data from an ongoing Phase II open-label extension study of its TTR-mediated amyloidosis therapy patisiran, which showed that the drug treatment was associated with a stabilization of neurological impairment progression in patients

NEW YORK (GenomeWeb) – RNAi startup Arcturus Therapeutics this week released new preclinical data on its lead transthyretin-mediated amyloidosis (ATTR) candidate LUNAR-101, showing that a single dose of the drug could cut levels of its target by more than 90 percent in non-human primates (NHPs) f

NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week announced that it has initiated a screening study, called DISCOVERY, to examine the prevalence of transthyretin (TTR) mutations in patients believed to have cardiac amyloidosis, in support of its continued development of an RNAi-based treat

NEW YORK (GenomeWeb) – Officials from Alnylam Pharmaceuticals last week provided updates on the two drug candidates from the company's flagship transthyretin-mediated amyloidosis program, stating that the intravenously delivered agent patisiran is proceeding toward a possible market approval in t

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