Rare diseases | pharmaphorum

Saturday 7 February 2026

Rare diseases

News

Jeffrey Dayno of Harmony Biosciences

Harmony buys cannabidiol firm Zynerba in $200m deal

Neuroscience specialist Harmony Biosciences has agreed to acquire Zynerba, adding its cannabidiol (CBD) gel product Zygel for Fragile X syndrome and other rare neuropsychi

News

rare disease patients

LifeArc rare disease programme to invest £100m improving pat...

Self-funded, non-profit medical research organisation and charity LifeArc has announced today that it is to launch the Rare Disease Translational Challenge, which will inv

R&D

AI breakthrough

AI could provide breakthrough for early diagnosis of rare di...

For people living with rare diseases, one of the major challenges is being able to receive a rapid and accurate diagnosis.

R&D

rare diseases

Sponsored

Trials and tribulations of drug development: What does it ta...

Rare diseases are a complex and challenging area of medicine, affecting millions of people worldwide.

R&D

strategy planning

Making accessibility strategy part of your rare disease tria...

Over the past several years, diversity, equity, and inclusion (DEI) has become a central subject of conversation in clinical trials.

Patients

pharmaphorum podcast episode 81

Challenges and opportunities with plasma-derived therapies

This newest pharmaphorum podcast welcomes Takeda's Dr Kristina Allikmets.