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FDA approves UCB’s Rystiggo to treat generalised myasthenia gravis

The rare autoimmune disease has a global prevalence of 100 to 350 cases per every one million people

- PMLiVE

Pfizer’s Talzenna combination receives FDA approval for metastatic prostate cancer

Up to 20% of patients develop metastatic prostate cancer within five to seven years of diagnosis

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Astellas and Cullgen announce protein degrader partnership worth up to $1.9bn

The companies will use Cullgen’s protein degradation platform to discover multiple candidates

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FDA approves Blueprint Medicines’ Ayvakit for indolent systemic mastocytosis

Ayvakit is now the first and only approved medicine designed to treat the underlying cause of the disease

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Astellas Pharma’s non-hormonal menopause drug granted FDA approval

Vasomotor symptoms affect up to 80% of menopausal individuals in the US

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NHS England announces rare genetic disorder fast-tracking service

Patients with inherited white matter disorders will benefit from earlier diagnosis and specialist care

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Pfizer and Astellas’ Xtandi shows promise in phase 3 prostate cancer study

The treatment is already approved in the US for three disease states of prostate cancer

- PMLiVE

Results for Astellas’ non-hormonal menopause drug published in The Lancet

Symptoms such as hot flashes and night sweats are the most common symptoms of menopause

Rare Diseases on Medscape Education: Pustular Psoriasis

Rare Disease Day may be over but our committment to advancing outcomes for patients continues. Watch this video to understand a patient's journey with pustular psoriasis.To learn more about Medscape's rare...

Medscape Education Global

Rare Diseases on Medscape Education: Long-Chain Fatty Acid Oxidation Disorder

Rare Disease Day may be over but our committment to advancing outcomes for patients continues. Listen to a mother explain her daughter's journey with a long-chain fatty acid oxidation disorder.To...

Medscape Education Global

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AstraZeneca announces major expansion of Canadian research footprint

The investment includes the creation of 500 jobs and a new rare disease research hub

- PMLiVE

Rare Disease Day 2023 – raising awareness around the world

Around one in 17 people will be affected by a rare disease at some point in their lives

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