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Zolgensma

- PMLiVE

Novartis publishes final positive phase 3 results of Zolgensma in Nature Medicine

Almost all children involved with copies of the SMN2 gene treated pre-symptomatically met key milestones

- PMLiVE

Novartis announces promising five-year data for leukaemia treatment Kymriah

Results have shown durable remission and long-term survival in children and young adults with B-cell acute lymphoblastic leukaemia

- PMLiVE

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

- PMLiVE

Genentech’s Evrysdi gets extended FDA approval for spinal muscular atrophy

The drug is now approved to treat SMA in children and adults of all ages

- PMLiVE

Novartis’ Kymriah receives FDA approval to treat follicular lymphoma

The CAR-T cell treatment is approved for adult patients who have had two or more previous therapies

- PMLiVE

Novo Nordisk and Flagship Pioneering to collaborate on new research programmes

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

- PMLiVE

EC approves Novartis’ Kymriah for advanced blood cancer

Kymriah is the first CAR-T cell therapy of its kind approved in the EU for follicular lymphoma

- PMLiVE

Novartis restructures organisation to support growth and innovation

The new operational structure will involve integrating pharmaceuticals and oncology business units into an Innovative Medicines business

- PMLiVE

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

- PMLiVE

Novartis’ Kesimpta trial evaluates treatment of COVID-19 in MS patients

There are an estimated 130,000 people with MS in the UK

- PMLiVE

FDA approves Novartis’ Pluvicto for prostate cancer

The treatment is the first FDA approved targeted radioligand therapy for mCRPC patients

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

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