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Batten disease

Amicus shares early look at Batten disease gene therapy

Fierce Biotech

Thu, 08/1/19 - 10:31 am

Amicus Therapeutics

Amicus Therapeutics Ramps Up Gene Therapy Portfolio with Acquisition of 10 Programs

CP Wire

Thu, 09/20/18 - 09:46 am

Amicus Therapeutics

BioMarin CEO says 'the system will have to determine' the worth of its new $702,000 drug

CNBC

Sun, 04/30/17 - 12:51 pm

Rare feat: BioMarin rewrites playbook for small-market drug approvals

Bizjournals.com

Tue, 01/27/15 - 09:41 am

StemCells announces results of long-term follow-up study in Batten disease

Yahoo/Fly on the Wall

Mon, 10/21/13 - 09:17 am

BioMarin doses first patient in Phase 1/2 trial with BMN 190

Yahoo/Fly on the Wall

Mon, 09/23/13 - 10:48 am

BioMarin submits CTA for BMN-190 for Batten disease

Yahoo/Fly on the Wall

Mon, 04/1/13 - 10:59 am

StemCells shelves Batten disease program — not enough patients for trial

bizjournals.com

Sat, 04/9/11 - 10:55 am