Rare diseases

News

FDA delays decision on Roche's SMA drug to review extra data

The FDA has extended its review of Roche’s spinal muscular atrophy (SMA) drug risdiplam after the pharma submitted additional data.

News

Novartis publishes long-term data from SMA gene therapy

Novartis has produced data demonstrating its Zolgensma gene therapy produces long term benefits in the rare muscle wasting disease spinal muscular atrophy – information that will be closely

Views & Analysis

Genetic testing hopes to speed up drug development in rare e...

When it comes to fighting rare diseases, the biggest barrier to overcome is often the lack of knowledge around the condition.

Views & Analysis

Understanding the impact of a rare disease – the Health Hero...

Episode one of Kantar’s Health Heroes podcast looks at caregiving challenges for Gaucher disease and how patient registries can help deliver new treatment innovations and better outcomes fo

AI in Pharma Marketing

Genetic engineeering concept with 3d rendering robotic hand editing dna helix

AI for rare diseases: putting tech to work for small populat...

With the 12th annual Rare Disease Day on the horizon, it’s a pertinent time to think about how one of the most exciting technological innovations of the last decade, artificial intelligence

Views & Analysis

The biggest challenges in rare disease drug development – and how the industry is fighting them

The biggest challenges facing rare disease pharma – and how ...

As Rare Disease day 2020 approaches, we take a look at the biggest challenges facing orphan drug developers and ask whether the future is bright or bleak for these difficult conditions.

FDA delays decision on Roche's SMA drug to review extra data

Novartis publishes long-term data from SMA gene therapy

Genetic testing hopes to speed up drug development in rare e...

Understanding the impact of a rare disease – the Health Hero...

AI for rare diseases: putting tech to work for small populat...

The biggest challenges facing rare disease pharma – and how ...

Relief for AZ as Truqap hits the spot in prostate cancer

Coming soon: The Life Sciences Industry Report 2025 – insigh...

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