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CHAPLE disease

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Sandoz receives FDA approval for first denosumab biosimilars Wyost and Jubbonti

The biosimilars have been authorised for all indications covered by Amgen's reference medicines

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Novartis shares positive results for spinal muscular atrophy gene therapy Zolgensma

The company said the results support the use of the one-time therapy in older and heavier children

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Johnson & Johnson’s Rybrevant granted FDA approval for lung cancer indications

The drug was previously authorised for use by the US regulator under the accelerated approval pathway

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FDA awards Orphan Drug Designation to SynaptixBio for rare brain disease therapy

Isolated hypomyelination is a form of TUBB4A leukodystrophy that has been identified relatively recently

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Radboud researchers use new method to improve diagnosis of rare diseases

Approximately 7,000 rare diseases collectively impact up to 5.9% of the global population

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OneChain-led consortium granted €1.9m to advance CAR-T therapy for rare leukaemia

Acute lymphoblastic leukaemia has an incidence of around 1.5 cases per 100,000 people

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Clinigen launches ‘What is Possible?’ campaign on Rare Disease Day to accelerate access to medicines

There are over 7,000 rare diseases impacting more than 300 million people globally

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FDA approves Allecra’s combination antibiotic Exblifep for complicated UTIs

Complicated UTIs account for approximately 19% of all healthcare-associated infections

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Novo Nordisk and Neomorph enter molecular glue degrader partnership worth over $1.4bn

The companies will work on therapies for cardiometabolic and rare diseases

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FDA approves Alvotech and Teva’s Simlandi as interchangeable Humira biosimilar

Simlandi is approved to treat multiple indications, including rheumatoid arthritis and Crohn’s disease

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Alexion launches ‘colourUp4RARE challenge’ to mark Rare Disease Day 2024

The campaign is aimed at raising awareness of how to improve quality of life for patients

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Johnson & Johnson’s Tecvayli receives FDA approval for reduced dosing in multiple myeloma patients

More than 35,000 cases of the blood cancer are expected to be diagnosed in the US this year

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