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CROs and trial design
Early access and regulation
Value demonstration
Strategic communications
CROs and trial design
Shire links with US hospital for rare disease research
Willwork with Cincinnati Children's Hospital Medical Centre novel therapies
FDA awards $19m in rare disease grants
US regulator supports 15 orphan drug projects
The moving parts of orphan drug development
Tackling rare diseases requires high levels of cooperation, regulatory support and a determination to succeed
Shire boosts rare disease portfolio with $260m Lumena deal
Acquisition of US company will bring two mid-stage candidates for rare liver diseases
Amgen cholesterol antibody shines in rare disease trial
Evolocumab passes late-stage study
WCCT Global talks up orphan drug benefits of new service
CRO's In-Site programme will harness digital technology for patient recruitment
Sanofi strikes $700m rare diseases deal
Company's Genzyme division will obtain significant rights to Alnylam’s pipeline of orphan drugs
Novartis myeloma drug clears phase III trial
Panobinostat on course to be first in new class of treatment for bone marrow cancer
Shire snaps up Viropharma to build $2bn rare disease unit
Gains access to Cinryze in expensive but 'strategically sound' deal
GSK wins anthrax contract from US government
Signs $196m deal for its monoclonal anti-toxin raxibacumab
Chiesi acquires Zymenex and mulls new rare disease unit
Deal adds late-stage orphan drug to Italian pharma company's pipeline
Shire extends rare genetic disease alliance
Agrees option with Santaris to nominate additional discovery targets
EC sets up expert group on rare diseases
Will revamp EUCERD to better handle development of orphan medicines
Boehringer: clinical trial set-up poses challenge in rare diseases
Says more support needed in regulatory process for orphan drugs
Rare diseases among initial priorities of UK DNA mapping body
Genomics England will also help advance research into cancer and infectious diseases
Healthcare at Home and QED partner on orphan diseases
OrphanReach alliance will see companies provide product lifecycle services
Roche and Isis in $392m deal to research drugs for brain disorder
Will develop antisense drugs for Huntington's disease
Santhera's Raxone turned down for rare eye condition
EMA concerned about drug's ability to improve eyesight
PatientsLikeMe takes aim at one of world's rarest diseases
Creates open registry and community for alkaptonuria patients
Pfizer acquires spinal muscular atrophy research programme
Gains rights to drug candidate RG3039 in deal with Repligen that could be worth up to $70m
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