Rare Disease Day 2021

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

BeiGene’s Brukinsa gets green light from NICE as Waldenström’s macroglobulinemia treatment

The condition occurs in less than 2% of patients with non-Hodgkin lymphomas

bluebird bio’s Skysona approved by FDA to treat cerebral adrenoleukodystrophy

The gene therapy was shown to slow the progression of neurologic dysfunction

bluebird bio’s beta-thalassaemia gene therapy receives FDA approval

Patients with the most severe form of the blood disorder develop life-threatening anaemia and have to receive regular blood transfusions

bluebird bio reveals plans to launch two gene therapies

Both therapies were endorsed by the US FDA Cell Tissue & Gene Therapy Advisory Committee

EFPIA-EURORDIS joint statement published on patient access to medicines for rare diseases

The statement contains a series of proposals to improve the lives of people living with rare diseases by addressing access barriers to transformative medicines

Novo Nordisk and Flagship Pioneering to collaborate on new research programmes

The companies will focus on rare and cardio-metabolic disease areas

bluebird bio maintains its focus on gene therapy opportunities during structural changes

The company plans to reduce its workforce amid efforts to reduce its operating costs

Nucleus X Consulting – where patient engagement meets strategic consultancy

Nucleus X Consulting consists of an agile team of specialists that combine expertise in all aspects of patient engagement along with strategic consultancy in rare disease and early-phase medical commercialisation.We...

Nucleus Global

Extraordinary lives: advancing change in rare diseases (Part 2)

In support of Rare Disease Day, we’re excited to launch part 2 of our video series; Extraordinary lives: advancing change in rare diseases.Why is raising awareness of rare diseases so...

Lucid Group Communications Limited

Nucleus Global launches Nucleus X Consulting, a specialist consultancy with deep expertise in patient engagement, rare disease, and early-phase medical commercialization

Nucleus Global, the largest specialist medical communications network in the world, announces the launch of a new consultancy to add to its family of agencies. Nucleus X Consulting consists of...

Nucleus Global

Extraordinary lives: advancing change in rare diseases (Part 1)

Rare Disease Day is all about raising awareness and generating change for the 300 million people worldwide living with a rare disease. The global rare disease community and its supporters come together on...

Lucid Group Communications Limited

bluebird bio shares therapy updates for 2022

The company provided information for its first two gene therapies and its lovo-cel BLA submission for sickle cell disease

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Integrated Care Specialist - North & West Yorkshire

Territory Sales Manager - Yorkshire/North East

Territory Sales Manager - North London & East Anglia

Key Account Manager - Urology - East Anglia, Beds & Bucks

Territory Sales Manager - Ostomy Care - S Central/SW England

Territory Sales Manager - Ostomy Care - Wales, South Mids