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- PMLiVE

Chiesi

- PMLiVE

FDA gives priority review status to BMS/ AZ’s metreleptin

Former obesity prospect has potential in the rare disease lipodystrophy

- PMLiVE

Orphan status in US for Astellas/ Basilea antifungal

Isavuconazole is being developed as a treatment for invasive aspergillosis

- PMLiVE

FDA gives Alexion’s metabolic drug breakthrough status

Reduces requirements for registration, potentially speeding up approval

Healthcare at Home and QED partner on orphan diseases

OrphanReach alliance will see companies provide product lifecycle services

Payer focus in the personalised world of oncology and orphan diseases

Personalised medicine is transforming disease understanding and many traditional diagnoses may be fundamentally revised based on the new scientific understanding

- PMLiVE

FDA approves drugs for rare genetic liver condition and to reverse anticoagulation

Raptor’s Procysbi and CSL Behring’s Kcentra recommended in US

Rare disease patients face 5-7 year wait for diagnosis

Doctors often lack time and resources to manage patients

Roche - Basel

Roche and Isis in $392m deal to research drugs for brain disorder

Will develop antisense drugs for Huntington's disease

- PMLiVE

Novartis provides multimedia support to Rare Diseases Day

Collaborates with Eurordis on multimedia education campaign

- PMLiVE

Shining a light on orphan medicines

Orphan diseases represent one of the most exciting and emotive areas in modern medicine and need a communications strategy that covers a spectrum of issues

Lonafarnib: promising results in first-ever trial for progeria

Treatments for progeria are limited but lonafarnib and FTIs as a class have given renewed hope for children with the condition

- PMLiVE

PatientsLikeMe takes aim at one of world’s rarest diseases

Creates open registry and community for alkaptonuria patients

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