regeneron headquarters

Regeneron’s Veopoz granted FDA approval for ultra-rare immune disease

Fewer than ten patients in the US have been diagnosed with CHAPLE disease

- PMLiVE

AstraZeneca’s rare disease unit to acquire Pfizer’s early-stage gene therapies for $1bn

The transaction could potentially see staff associated with the portfolio move to Alexion

- PMLiVE

Astellas signs $962m rare eye disease deal with 4D Molecular Therapeutics

The company will gain the rights to use 4DMT’s gene therapy vector for at least one target

- PMLiVE

LifeArc launches rare disease programme with £100m investment

An initial £40m will go towards the creation of up to five rare disease research centres

- PMLiVE

NIHR and MRC establish UK Rare Disease Research Platform with £14m investment

The platform brings together expertise from across the UK rare disease research system

- PMLiVE

To support patient-centricity in clinical trials, look to rare disease communities

How patients are seeking new treatment options that will advance the care of their communities

Biogen Idec building

Biogen’s Qalsody granted FDA accelerated approval for rare form of ALS

Mutations in the SOD1 gene are responsible for approximately 2% of all cases

- PMLiVE

NHS England announces rare genetic disorder fast-tracking service

Patients with inherited white matter disorders will benefit from earlier diagnosis and specialist care

- PMLiVE

Rare diseases: not so rare after all

The brave new world addressing rare diseases – a way ahead to better and more inclusive treatment

- PMLiVE

Rare Disease Day 2023 – raising awareness around the world

Around one in 17 people will be affected by a rare disease at some point in their lives

- PMLiVE

UK government announces new rare diseases action plan for England

The framework lists 13 actions that have been developed with the rare disease community

- PMLiVE

SMC enables access to Kyowa Kirin’s Crysvita for adults with rare genetic disease

XLH is a life-long genetic disease that causes abnormalities in the bones, muscles and joints

- PMLiVE

FDA gives orphan drug status to Avidity’s rare muscular dystrophy treatment

There are currently no approved treatments for patients living with the disease

- PMLiVE

Kite reports positive three-year results for CAR T-cell therapy in rare blood cancer

Around 790 people are diagnosed with acute lymphoblastic leukaemia each year in the UK

- PMLiVE

Roche reports positive results for crovalimab as rare blood disease treatment

The drug was shown to be as effective as current treatment, with less frequent dosing