Subscribe to
this category


A total of 208 posts are filed under Research
Abeona Therapeutics Receives Regulatory Approval to Initiate Clinical Trial in Australia with ABO-102 Gene Therapy For Patients with MPS IIIA
Clinical Trial Notification (CTN) Enables Initiation at 3rd Global Site and Accelerates Enrollment in the ABO-102 MPS IIIA Gene Therapy… Continue Reading
Introducing SideEQ: A Free Tool That Helps You Manage Cancer Treatment Side Effects
SideEQ is a free, interactive platform which enables people living with cancer to track and manage their side effects so they can improve… Continue Reading
ProQR to Present its Ophthalmology Pipeline at ARVO, Highlighting Programs in Leber’s Congenital Amaurosis and Usher Syndrome
ProQR Therapeutics N.V.(Nasdaq:PRQR) today announced that the Company will present three abstracts during the ARVO conference, including… Continue Reading
Powered by CIRM Grant, jCyte Launches New Clinical Trial for RP
Cell therapy company jCyte is launching a phase 2b clinical trial to test the safety and efficacy of its developmental retinitis pigmentosa… Continue Reading
Teva Showcases CNS Portfolio at 69th Annual Meeting of the American Academy of Neurology
Teva Pharmaceutical Industries Ltd., (NYSE and TASE: TEVA) today announced data for five of the Company’s innovative therapies for central… Continue Reading
Horizon Pharma plc Announces FDA Approval to Expand the Age Range for RAVICTI® to People with Urea Cycle Disorders Ages Two Months +
Horizon Pharma plc (NASDAQ:HZNP), a biopharmaceutical company focused on improving patients’ lives by identifying, developing, acquiring… Continue Reading
Promising Week for Treatment of Form of Batten Disease: Approvals Granted by FDA and Europe’s CHMP
Significant progress was made last week for those suffering from Batten Disease (CLN2), a form of lysosomal storage disorder characterized… Continue Reading

Follow us on Twitter

Upcoming Events

Sep 14

RARE Patient Advocacy Summit

September 14 @ 8:00 am - 5:30 pm
Sep 14

RARE Partnering 2017

September 14 @ 8:00 am - 5:00 pm
Sep 15

RARE Patient Advocacy Summit

September 15 @ 8:00 am - 4:30 pm
Sep 15

RARE Champion of Hope Awards

September 15 @ 5:00 pm - 9:30 pm
Sep 16

Corporate Alliance

September 16 @ 9:00 am - 12:00 pm
Sep 16

RARE Day of Beauty

September 16 @ 12:00 pm - 5:00 pm
Sep 16

Tribute to Champions of Hope

September 16 @ 5:30 pm - 10:30 pm