Batten disease

Amicus shares early look at Batten disease gene therapy
Fierce Biotech
Thu, 08/1/19 - 10:31 am
Amicus Therapeutics
Batten disease
gene therapy
AAV-CLN6
Amicus Therapeutics Ramps Up Gene Therapy Portfolio with Acquisition of 10 Programs
CP Wire
Thu, 09/20/18 - 09:46 am
Amicus Therapeutics
gene therapy
Batten disease
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
Biomarin
Brineura
drug pricing
Batten disease
Rare feat: BioMarin rewrites playbook for small-market drug approvals
Bizjournals.com
Tue, 01/27/15 - 09:41 am
Biomarin
orphan drugs
BMN-190
Batten disease
StemCells announces results of long-term follow-up study in Batten disease
Yahoo/Fly on the Wall
Mon, 10/21/13 - 09:17 am
StemCells
stem cells
Batten disease
HuCNS-SC cells
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
BMN-190
Batten disease
BioMarin submits CTA for BMN-190 for Batten disease
Yahoo/Fly on the Wall
Mon, 04/1/13 - 10:59 am
Biomarin
BMN-190
Batten disease
StemCells shelves Batten disease program — not enough patients for trial
bizjournals.com
Sat, 04/9/11 - 10:55 am
StemCells
Batten disease