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LEO Pharma acquires rights for dersimelagon from Tanabe Pharma

The company will pay $435m in up front and near-term milestone payments

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Sanofi announces phase 3 study data for infantile-onset Pompe disease

Pompe disease is a rare, progressive neuromuscular disease, with infantile onset being the most aggressive form

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Sanofi’s Cenrifki approved in Europe for secondary progressive MS without relapses

SPMS is a debilitating stage of MS where patients experience increasing levels of disability

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Sanofi appoints Paulo Fontoura as Global Head of R&D

Fontoura previously held senior leadership roles at Xaira Therapeutics and Roche

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Sanofi’s Sarclisa approved by MHRA as UK’s first anti-cancer treatment given via on-body injector

It is the first anticancer treatment in the UK and globally to be administered this way and a first in multiple myeloma

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LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

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Sanofi’s Tzield gets expanded FDA approval for early-stage type 1 diabetes

The treatment can be used to delay the onset of clinical type 1 diabetes

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Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

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PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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New report identifies stigma as a structural barrier in rare disease

The study finds that healthcare settings are a leading source of stigma for people living with rare conditions

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UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

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Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

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