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NIH

- PMLiVE

US FDA launches ultra-rare diseases framework

The draft guidance specifically discusses genome editing and RNA-based therapies

- PMLiVE

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

phthalmologist, optometrist checking child, little girl vision looking for problems and caring for eye

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

- PMLiVE

PureTech’s deupirfenidone gets FDA and EC Orphan Drug Designations for idiopathic pulmonary fibrosis

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or fewer than five in 10,000 individuals in the European Union

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Diabeloop’s DBLG2 receives FDA and CE clearance for automated insulin delivery without meal input

While a lack of meal announcements can lead to lower glycaemic control, it reduces the mental burden for those with type 1 diabetes

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#ShortenTheJourney

For the millions of people living with a rare disease, the path to a diagnosis (if ever received) is often a long and frustrating odyssey. This delay is frequently driven...

Medscape Education

- PMLiVE

Johnson & Johnson’s Darzalex Faspro regimen receives FDA approval for multiple myeloma

Multiple myeloma has an average five-year survival rate of 59.8% and is currently incurable

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Novartis’ ianalumab receives FDA Breakthrough Therapy designation for Sjögren’s disease

Approximately half of those with Sjögren’s disease are thought to be undiagnosed

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FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

- PMLiVE

FDA launches TEMPO digital health pilot

Digital technologies can improve remote patient care for those with chronic diseases

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Richard Pazdur, FDA’s drug evaluation chief, to leave FDA just weeks into new role

Pazdur was previously head of the FDA’s Oncology Center of Excellence

- PMLiVE

Merck KGaA given FDA Fast Track designation for generalised myasthenia gravis drug

Cladribine capsules could become the first ever oral treatment for gMG

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