First‑in‑class innovation
LEO Pharma acquires rights for dersimelagon from Tanabe Pharma
The company will pay $435m in up front and near-term milestone payments
LEO Pharma to acquire Replay gene therapy platform
The acquisition will advance innovation for patients with rare skin diseases
Shionogi starts phase 2 clinical trial for Pompe Disease
The rare disease affects around one in every 22,000 people worldwide
PharmaQuest returns: EPA perspectives, through a rare disease lens
By Lottie Sequerra and Emily Morton-West
UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency
TK2d is an extremely rare disease characterised by severe and progressive muscle weakness
Sanofi announces positive results for venglustat in type 3 Gaucher disease
There are currently no approved treatments for the neurological symptoms of this disease
ISPOR’s 2026–2027 HEOR Trends report: The Era of Evidence Judgment
In response to ISPOR’s recently published 2026-2027 HEOR Trends, David Miller, CEO, Genesis Research Group, suggests that we’re no longer being evaluated on our ability to produce data, but on...
Havas Life London unveils AI awareness campaign
The campaign highlights the dangers of AI misinformation in rare disease
FDA approves first gene therapy for Wiskott-Aldrich syndrome
Symptoms of the rare disease include bleeding, eczema and frequent infections
AstraZeneca announces $2bn investment in US manufacturing
The new and expanded facilities will support production of rare disease drug treatments
AstraZeneca reports positive results from rare disease trial
HES is a group of rare disorders characterised by persistently elevated levels of white blood cells
Genesis Research Group contributes strong scientific program to ISPOR Europe and warmly invites visitors to booth 212
Genesis Research Group is proud to be attending ISPOR Europe 2025 in Glasgow from 10-12th November with a strong scientific program that includes an HEOR Theater session, contributions to 10...