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Handl Therapeutics

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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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UCB releases new data on Bimzelx for hidradenitis suppurativa

The disease affects around 1% of the population in most studied countries

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Lilly and MeiraGTx partner in $475m ophthalmology deal

The companies will develop and commercialise therapies for genetic conditions

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UCB’s Fintepla shows positives results for Lennox-Gastaut syndrome

The study demonstrated a sustained reduction in seizures for patients

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UCB shares promising results for fenfluramine in ultra-rare form of epilepsy

CDKL5 deficiency disorder occurs in approximately one in 40,000 to 60,000 live births

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UCB’s Bimzelx demonstrates lasting efficacy in phase 3 psoriatic arthritis trials

Approximately 125 million people worldwide are affected by some form of psoriasis

The (clinical) trials and tribulations of gene therapy – why meaningful patient engagement strategies matter

By Sarah Mohamad and Lauren Coleman-Bennett

Amiculum

What do people look for in gene therapy materials? Results from our survey of patients and relatives

By Sam Fraser

Amiculum

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CSL Behring announces four-year results for haemophilia B gene therapy Hemgenix

The genetic bleeding disorder affects an estimated one per every 19,283 male births in the US

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NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease

About 15,000 people in England are living with the inherited blood disorder

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UCB’s Bimzelx granted FDA approval to treat hidradenitis suppurativa in adults

The inflammatory skin disease affects approximately one in every 100 people in the US

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UCB shares ‘encouraging’ data for Alzheimer’s disease candidate bepranemab

The drug showed benefits across key secondary endpoints, including tau accumulation

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