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Novartis’ Gene Therapy Luxturna Gets Routine Funding In Scotland

Scotland’s health technology assessment body has granted routine reimbursement to Novartis’ vision loss drug Luxturna after an interim funding period via its ultra-orphan pathway, while Chiesi’s Filsuvez is set to begin its journey on the same pathway.

Europe United Kingdom

Keeping Track: Return & Renewal At US FDA

Takeda’s TAK-721 is back, ARS Pharma will be back soon to appeal neffy complete response letter, US FDA’s Project Renewal brings Temodar labeling in current day, and more highlights from the Pink Sheet’s US FDA Performance Tracker.

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Crinetics’ Paltusotine Future Appears Bright With First Phase III Acromegaly Readout

The once-daily oral somatostatin receptor type 2 (SST2) agonist outshined placebo in patients switching from standard of care, offering an attractive alternative to once-monthly injectables.

Clinical Trials Rare Diseases

Paradigm Plots Revival Of Butterfly Skin Drug After Amicus Deal

Chiesi-owned Amryt and Krystal Biotech have approved treatments for epidermolysis bullosa but Paradigm CEO Robert Ryan tells Scrip that SD-101, a topical cream he helped develop over 20 years ago, has the potential to be offer a far more extensive and efficacious solution for many more sufferers.   

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