aromatic amino acid decarboxylase deficiency
FDA approves Priovant’s Lisraya for dermatomyositis
This is the first oral drug treatment for this rare disease in adults
Lundbeck completes patient randomisation for phase 3 trial for multiple system atrophy treatment
The rare neurodegenerative disorder has no cure and no approved treatments
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...
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.
#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...
Women Leaders Rewriting the Protocols of Rare Disease Care
The true measure of a medical leader is the courage to act when existing systems fail the patient. This is the professional and personal ethos of clinical immunologist Dr. Hilary...
Medscape Wins Anthem Award for Rare Disease Education Platform
Platform has reached 2.4 million healthcare professionals worldwide since 2021. Newark, NJ - [November 18, 2025] - Medscape, the leading digital platform for physicians and healthcare professionals (HCPs), has been...
Lilly and MeiraGTx partner in $475m ophthalmology deal
The companies will develop and commercialise therapies for genetic conditions
Medscape Education Wins SILVER for Rare Disease Campaign
We are thrilled to announce that Medscape Education has been honored with the SILVER Award in the Patient Education Information division category by the 2025 National Health Information Awards! This...
Insights From ISPOR 2025: Breaking the Data Barrier in Rare Diseases – Can AI Bridge the Gap?
Last month, the ISPOR annual meeting in Montreal was brimming with opportunities to explore the latest developments and insights in health economics and outcomes research (HEOR) and market access. In...
Stronger Together: Medscape Education Celebrates Accomplishments in Rare Disease and Anticipates the World Health Assembly Adoption of the Resolution on Rare Diseases
Medscape Education is proud to be showcasing the power of working together in rare disease education and advocacy by co-hosting a side event at the 78th World Health Assembly in...
The (clinical) trials and tribulations of gene therapy – why meaningful patient engagement strategies matter
By Sarah Mohamad and Lauren Coleman-Bennett