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Familial Amyloid Polyneuropathy (also known as transthyretin amyloidosis, TTR, and FAP) is a group of endangering multisystem disorders which are transmitted as an autosomal dominant trait. It is caused by the excessive accumulation of amyloid in the organs and tissues. The first identified cause of FAP is the Val30...

Gene therapy is back into play after a decade-long failed deliveries. 2017 was a golden year in gene therapy arena, various companies are aggressively developing gene therapy based treatments targeting life-threatening indications. Spark therapeutics got USFDA approval for the first-ever gene therapy based blindness...

Hereditary Angioedema (HAE) is an autosomal dominant disease which is caused by a deficiency in functional C1 inhibitor. It is a rare hereditary disease which is characterized by painful, unpredictable, recurrent attacks of inflammation and swelling affecting the hands, feet, face, abdomen, urogenital tract, and the...

British scientists looking forward to treat Alzheimer’s disease through diabetes drug A group of British scientists are looking for the treatment of Alzheimer’s disease in an unlikely source i.e. a drug used for treating Type 2 Diabetes. The team, from Lancaster University, has reported that an experimental three-p...

AbbVie aims for best-in-class psoriasis prospect The last few years have seen a wave of new biologics take the psoriasis market by storm. But a best-in-class product may still be on its way. AbbVie prospect risankizumab showed it could bring 47% of patients to complete skin clearance by week 16 of treatment in a pha...

KINECT 4 Phase III Study Demonstrating INGREZZA® Improves Tardive Dyskinesia Symptoms Neurocrine Biosciences, Inc. announced new long-term data from the KINECT 4 Phase III open-label study demonstrate that once-daily INGREZZA® (valbenazine) capsules, the first FDA approved treatment for adults with tardive dyskinesi...

Cluster of differentiation 40 or CD40 is a co-stimulatory protein found on the antigen presenting cells of the immune system. Agonistic CD40 molecules which include monoclonal antibodies (mAb) offer a new therapeutic option with the potential to generate anticancer immunity by various mechanisms. CD40 is a TNF recep...

EMA to relocate to Amsterdam by March 2019 Results of the Monday's vote for the European Medicines Agency relocation led to the emergence of Amsterdam as the new headquarters, beating the other finalists Milan and Copenhagen. Nineteen European Union member states submitted proposals for the EMA headquarters, and Mil...

Novartis ventures itself into developing new editing technology Novartis has moved beyond CRISPR and CAR-T gene editing technology with its latest deal with Homology Medicines, aiming to improve upon its R&D work in the arena of cell and gene therapy. The deal involves Novartis using the gene editing technology ...

Takeda and Portal Instruments partner to bring about needle-free form of drug delivery Takeda Pharmaceutical and Portal Instruments have partnered with a deal worth $100 million to develop a needle-free drug delivery device and increase patient compliance rates for biologic medicines manufactured by the former compa...