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Bristol Myers Squibb’s New Drug Application for mezigdomide accepted by FDA

Mezigdomide improves progression-free survival in patients with relapsed or refractory multiple myeloma

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Teva submits FDA application for paediatric Tourette syndrome therapy

Ecopipam could become the first new FDA-approved treatment for paediatric Tourette syndrome in over ten years

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Roche’s Enspryng given FDA Priority Review as thyroid eye disease treatment

This is the first and only at-home subcutaneous treatment option for this disease

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Regeneron’s New Drug Application for cemdisiran accepted for review by FDA and EMA

Globally, around 150 to 200 out of every million people have myasthenia gravis

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Roche’s Tecentriq gets FDA Priority Review for specific stage 3 colon cancer

Over one million people are diagnosed globally each year and around 30% of stage 3 patients relapse within five years

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Lilly’s Ebglyss approved by FDA as maintenance dose for atopic dermatitis

This new option for patients allows for as few as six maintenance injections per year

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FDA gives Rare Paediatric Disease designation to Satellite Bio for urea cycle disorders

The mortality rates of UCDs exceeds 25%, with no curative treatment available for newborns

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BMS’ Opdivo gets expanded FDA and EU approval for Hodgkin lymphoma

This type of cancer is the most common one diagnosed in teenagers

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Lundbeck completes patient randomisation for phase 3 trial for multiple system atrophy treatment

The rare neurodegenerative disorder has no cure and no approved treatments

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US FDA launches ultra-rare diseases framework

The draft guidance specifically discusses genome editing and RNA-based therapies

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Data, Evidence, Education: Tackling the Global Multi‑Trillion‑Dollar Rare Disease Burden

Rare diseases are individually uncommon, yet together they affect an estimated 300 million+ people worldwide and touch the lives of over one billion when families and caregivers are included. For...

Medscape Education

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Why patient engagement must come first in rare disease trials

Explore why patient engagement is the cornerstone of successful rare disease trials, emphasising the importance of designing studies around the realities of patients' lives to build trust and improve outcomes.

Cuttsy + Cuttsy

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