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Researchers publish promising results for ultra-rare disease gene therapy method
Gene therapy method evaluated in seven children with aromatic amino acid decarboxylase deficiency
Rare diseases: why they can be so difficult to identify
Symptoms shared with more common diagnoses can make it more difficult to spot underlying rare diseases
NICE recommends Ultomiris for NHS use in patients with rare blood disorders
摘要是一个ultra-rare and life-threatening disease that causes progressive injury to vital organs
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
Value demonstration
Assessing drugs for ultra-rare conditions in the UK
Alexion’s Soliris has become the first drug to pass through NICE’s new highly specialised technology process
No price ceiling for rare disease drugs, say MPs
BIA survey comes ahead of consultation on evaluation framework for ultra-orphans
NICE and ‘ultra orphans’
NICE and its processes for ‘highly specialised therapies’
Early access and regulation
Another lost decade for research?
What does the patent landscape look like for antibiotics, vaccines and orphan drugs?
First von Willebrand disease therapy heads new FDA approvals
Baxalta's Vonvendi set for an early 2016 launch in the US
Wakix backed for European approval in narcolepsy
Bioprojet Pharma’s orphan drug given a positive opinion by CHMP
Strategic communications
Orphan drug firm Sobi opens new European base
Company'sBrussels office will also house its Benelux operations
Orphan drug market to reach $176bn by 2020
New report suggests market will almost double in five years
Channel strategy and orphan drugs
A plan for guiding decisions about a product's distribution channel is particularly relevant for orphan drugs, rare diseases or specialty medicines