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Rare Diseases
After Alfasigma licensing deal, Lynavoy picks up FDA nod
19 Mar 2026 //
FIERCE PHARMA
FDA approves leucovorin for Rare Genetic Disorder Causing Autism
11 Mar 2026 //
REUTERS
AbbVie, Gilead, GSK Support Annual Hepatitis Elimination Drive
29 Jul 2025 //
FIERCE PHARMA
GSK posts phase 3 itch data as it closes in on FDA approval
09 May 2025 //
PRESS RELEASE
Science 37 Doubles US Enrollment for GSK Phase 3 Rare Disease Trial
21 Nov 2024 //
GLOBENEWSWIRE
Sanofi gambles $80M on Fulcrum’s muscular dystrophy drug
14 May 2024 //
BIOPHARMADIVE
Benlysta granted Orphan Drug Designation by US FDA for systemic sclerosis
01 Feb 2023 //
PRESS RELEASE
First trial participant gets GSK`s 5-in-1 meningitis jab
19 Aug 2020 //
PHARMATIMES
Data suggest gene, cell therapy trial initiations in Europe lag behind
01 Nov 2019 //
ENDPTS
Roche puts down $75M to sign on AMD drug, following Huntington’s deal
11 Oct 2018 //
ENDPTS
Ionis may have found a home for dry AMD drug with Roche in $760M deal
10 Oct 2018 //
FIERCE BIOTECH
FDA nixes Akcea’s volanesorsen despite favorable AdComm
28 Aug 2018 //
FIERCE BIOTECH
Novartis farms out rare disease drug to LifeMax Labs
28 Aug 2018 //
FIERCE BIOTECH
GSK offloads rare disease gene therapies to Orchard Therapeutics
13 Apr 2018 //
FIERCE BIOTECH
NICE backs GSK’s €594k gene therapy for rare disease
04 Jan 2018 //
PMLIVE
GSK announces new priorities; plans to offload 130 non-core brands
02 Aug 2017 //
BIOSPECTRUM
GSK’s pipeline cull claims sirukumab, and possibly rare disease unit
28 Jul 2017 //
PMLIVE
GSK`s talk of selling out points up rare disease challenges
27 Jul 2017 //
FIERCE PHARMA
GSK gives up on rare diseases as gene therapy gets two customers
26 Jul 2017 //
REUTERS
Spark closes on first US gene therapy approval
18 Jul 2017 //
PMLIVE
Pfizer looks at building major gene therapy manufacturing facility in North Carolina
09 Mar 2017 //
FIERCE PHARMA
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