Can Servier’s $2.65bn Edgewise deal reshape the muscular dystrophy drug race?
Servier is buying sevasemten before pivotal muscular dystrophy data. The deal gives Edgewise cash and investors a sharper cardiology story.
Can Lilly turn Hanmi’s sonefpeglutide into the next long-acting GLP-2 rare disease bet?
Read why Lilly’s Hanmi sonefpeglutide deal could reshape long-acting GLP-2 therapy for short bowel syndrome.
Why Ono’s Phase 2 MSA data may matter beyond one World Parkinson Congress poster
Ono’s ONO-2808 data hints at disease modification in MSA. See why Phase 3 may decide whether the signal becomes a breakthrough.
Neurocrine’s $2.9bn Soleno deal raises the stakes in Prader-Willi syndrome treatment
Find out how Neurocrine’s $2.9bn Soleno acquisition could reshape VYKAT XR’s rare disease launch strategy.
Is iECURE’s infant gene editing therapy quietly changing the OTC deficiency playbook?
A rare disease gene editing signal is only the start. iECURE now has to prove ECUR-506 can sustain OTC deficiency control.
Angelini Pharma bets $4.1bn on rare neurology scale with Catalyst Pharmaceuticals
Angelini gains U.S. rare disease scale. Catalyst gets certainty, but Firdapse concentration keeps the real test ahead.
Arrowhead just unlocked Australia for REDEMPLO, but the bigger RNAi test starts now
Arrowhead has regulatory momentum in rare lipid disease. The harder test is turning REDEMPLO approval into durable global uptake.
Arrowhead’s plozasiran clears CHMP: what a positive opinion for FCS means for siRNA’s regulatory arc in Europe
Plozasiran clears CHMP but the real test is reimbursement. Europe’s HTA bodies will now decide whether clinical FCS diagnosis is enough.
Can Genentech’s Enspryng become the first approved treatment for MOGAD after the Phase III METEOROID win?
Genentech’s Enspryng cut MOGAD relapse risk by 68% in Phase III. Read what the data could mean for regulators, clinicians, and the rare disease market.
Drug Farm’s DF-003 shows early signal in ROSAH syndrome, but can ALPK1 inhibition hold up in pivotal testing?
Drug Farm’s DF-003 showed early Phase 1b benefit in ROSAH syndrome. Read what the ALPK1 data could change next.