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Rare Diseases
Rocket Pharma Announces $180 M Sale of Priority Review Voucher
28 Apr 2026 //
PHARMIWEB
JPM Day 3: Rare Disease In The Spotlight
15 Jan 2026 //
BIOSPACE
Rocket Pharma`s Kresladi BLA Resubmission Accepted By FDA
14 Oct 2025 //
PHARMIWEB
Rocket Aborts Mission To Get Rare Disease Gene Therapy Approved
03 Oct 2025 //
FIERCE BIOTECH
Rocket Gets FDA RMAT for RP-A601 Gene Therapy
17 Jul 2025 //
BUSINESSWIRE
Rocket Pharma Gets FDA IND Approval for RP-A701 in DCM
30 Jun 2025 //
BUSINESSWIRE
Children`s Hospital Announces Gene Therapy Results for Danon
18 Nov 2024 //
PR NEWSWIRE
Rocket Gets EU Orphan Status For RP-A601 In PKP2 Cardiomyopathy
29 May 2024 //
BUSINESSWIRE
Rocket Pharma to light up buildings again for Rare Disease Day
26 Feb 2024 //
ENDPTS
FDA delays PDUFA date for Rocket’s rare white blood cell disorder gene therapy
13 Feb 2024 //
ENDPTS
Rocket boosted by FDA alignment on pivotal gene therapy trial
13 Sep 2023 //
FIERCE BIOTECH
US FDA grants Fast Track & Orphan Drug designations to Rocket Pharma’s RP-A601
10 Jun 2023 //
PHARMABIZ
Rocket Receives FDA Fast Track and Orphan Drug Designations for RP-A601
08 Jun 2023 //
BUSINESSWIRE
Rocket Pharma re-ups rare disease awareness with Empire State Building light-up
24 Feb 2023 //
ENDPTS
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