Can Vertex turn its $10bn Crinetics deal into a rare endocrinology powerhouse?
Discover why Vertex is paying $10 billion for Crinetics and how Palsonify and atumelnant could reshape rare endocrine treatment.
Pharma & Biotech
Pharma & Biotech
Neurocrine builds long-term case for Crenessity in congenital adrenal hyperplasia
See how Neurocrine’s two-year Crenessity data may reshape long-term classic CAH care, steroid reduction and pediatric outcomes.
Features & Analysis
A rare disease policy shift just landed at the WHO. Could it change hemophilia care worldwide?
Find out how the WHO hemophilia resolution could reshape diagnosis, treatment access, prophylaxis and bleeding disorder care worldwide.
Pharma & Biotech
Why Recordati’s $12.4bn takeover bid could reshape Italy’s listed pharma landscape
Recordati’s $12.4bn buyout bid tests whether rare disease growth can justify one of Europe’s biggest pharma take-private deals.
Pharma & Biotech
Could Agios and Avanzanite turn PYRUKYND into Europe’s next rare blood disease launch test?
PYRUKYND has EU approval in thalassaemia. Now Agios and Avanzanite face Europe’s harder test: access, uptake and reimbursement.
Pharma & Biotech
Can Amylyx turn open-label AMX0035 signals into a credible Phase 3 Wolfram syndrome trial?
Amylyx has rare disease momentum, but open-label data are not destiny. AMX0035 now faces its real Wolfram syndrome test.
Pharma & Biotech
What BridgeBio Pharma, Inc.’s latest data signals for the future of targeted endocrine therapies
Explore how BridgeBio Pharma, Inc.’s Phase 3 CALIBRATE data could transform ADH1 treatment and reshape rare endocrine care. Read more now.
Pharma & Biotech
What Chiesi’s $1.9bn KalVista deal reveals about the next phase of rare disease consolidation
Rare disease M&A is shifting from science risk to launch execution. Chiesi’s KalVista deal puts oral HAE therapy at the center.
Pharma & Biotech
Sentynl Therapeutics licenses Progerinin for progeria as rare-disease strategy expands beyond Zokinvy
Sentynl Therapeutics Inc., the United States-based rare disease subsidiary of Zydus Lifesciences Limited, has entered into a licensing agreement with South Korean biotechnology company PRG S&T to develop the investigational molecule Progerinin (SLC-D011) for Hutchinson-Gilford Progeria Syndrome, an ultra-rare genetic disorder that causes accelerated aging in children. The therapy has received orphan drug designation from
Pharma & Biotech
What the EU’s Elfabrio dosing update means for Fabry disease treatment burden
EU approves monthly Elfabrio dosing for Fabry disease. Discover how reduced infusion frequency could reshape enzyme therapy and treatment burden.