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.
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.
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.
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.
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.
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.
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 […]
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.
Can Hemab Therapeutics’ sutacimig finally deliver the first preventive therapy for Glanzmann thrombasthenia after FDA breakthrough designation?
FDA grants Breakthrough Therapy Designation to Hemab Therapeutics’ sutacimig for Glanzmann thrombasthenia. Discover what this means for bleeding disorder treatment.
Why YolTech Therapeutics’ YOLT-202 data matters for the future of gene editing in Alpha-1 Antitrypsin Deficiency
Early YOLT-202 data from YolTech Therapeutics suggests gene editing could redefine Alpha-1 Antitrypsin Deficiency treatment. Read the analysis.