Helix launches Whole Exome+ diagnostic test for rare and unexplained genetic conditions
Helix launches Whole Exome+ combining exome, mitochondrial and chromosomal analysis for patients with rare and unexplained genetic conditions.
Medical Devices & Diagnostics
Pharma & Biotech
Ionis wins first FDA approval for Alexander disease. Can Zanvastro make an ultra-rare launch work?
Zanvastro finally gives Alexander disease a targeted therapy. Its next challenge is finding patients and funding a $1.14 million annual regimen.
Pharma & Biotech
Shionogi deepens ALS real-world data strategy after $2.5bn Radicava acquisition
Read how Shionogi’s NeuroBANK partnership could shape Radicava evidence, ALS trial design and real-world treatment research.
Pharma & Biotech
Could BMN 333 help BioMarin reset expectations in achondroplasia treatment?
Find out how BioMarin’s VOXZOGO and BMN 333 data could reshape rare bone disorder treatment as competition intensifies.
Pharma & Biotech
Could Crenessity move beyond 21-hydroxylase CAH after Neurocrine’s ENDO 2026 data?
Find out how Neurocrine’s Crenessity data could expand rare CAH treatment thinking while key clinical questions remain.
Pharma & Biotech
Soleno Therapeutics VYKAT XR data strengthens long-term case in Prader-Willi syndrome
Find out how Soleno Therapeutics’ VYKAT XR data could reshape hyperphagia treatment in Prader-Willi syndrome.
Pharma & Biotech
Why Mirum Pharmaceuticals and Incyte’s zilurgisertib data could reset expectations in FOP treatment
Read how Mirum and Incyte’s zilurgisertib Phase 2 data could reshape FOP treatment, FDA review risk and rare disease strategy.
Pharma & Biotech
Xeris wins key Keveyis patent allowance, but will it really slow generic pressure?
Find out how Xeris’ Keveyis patent allowance could reshape rare disease exclusivity, generic pressure and investor sentiment.
Pharma & Biotech
Why Blood Cancer United’s luveltamab move could change rare pediatric cancer access
Find out how Blood Cancer United’s luveltamab access move could reshape rare pediatric AML drug rescue models.
Pharma & Biotech
Johnson & Johnson’s IMAAVY data sharpen the FcRn strategy beyond myasthenia gravis
Find out how Johnson & Johnson’s IMAAVY data could reshape treatment strategy in warm autoimmune hemolytic anemia.