Researchers publish promising results for ultra-rare disease gene therapy method

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
Rare diseases: why they can be so difficult to identifySymptoms 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
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
Shire links with US hospital for rare disease researchWillwork with Cincinnati Children's Hospital Medical Centre novel therapies
FDA awards $19m in rare disease grants
FDA awards $19m in rare disease grantsUS regulator supports 15 orphan drug projects
The moving parts of orphan drug development
The moving parts of orphan drug developmentTackling 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
Assessing drugs for ultra-rare conditions in the UKAlexion’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
No price ceiling for rare disease drugs, say MPsBIA survey comes ahead of consultation on evaluation framework for ultra-orphans
NICE and ‘ultra orphans’
NICE and ‘ultra orphans’NICE and its processes for ‘highly specialised therapies’

Early access and regulation

Another lost decade for research?
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
First von Willebrand disease therapy heads new FDA approvalsBaxalta's Vonvendi set for an early 2016 launch in the US
Wakix backed for European approval in narcolepsy
Wakix backed for European approval in narcolepsyBioprojet Pharma’s orphan drug given a positive opinion by CHMP

Strategic communications

Orphan drug firm Sobi opens new European base
Orphan drug firm Sobi opens new European baseCompany'sBrussels office will also house its Benelux operations
Orphan drug market to reach $176bn by 2020
Orphan drug market to reach $176bn by 2020New report suggests market will almost double in five years
Channel strategy and orphan drugs
Channel strategy and orphan drugsA plan for guiding decisions about a product's distribution channel is particularly relevant for orphan drugs, rare diseases or specialty medicines