Signals
33AL amyloidosis therapy reshapes around daratumumab and emerging amyloid-targeted programs
Daratumumab plus bortezomib first-line, isatuximab follow-on programs, and emerging anti-amyloid antibody therapy are restructuring AL amyloidosis management.
Neurotrophic keratitis acquires recombinant nerve growth factor therapy
Cenegermin (recombinant nerve growth factor) maturity, follow-on programs, and structured corneal-care pathways are reshaping neurotrophic keratitis management.
Lung cancer screening pathway integration matures
Low-dose CT screening adoption growth, AI-assisted nodule management, blood-based biomarker integration, and primary-care pathway formalisation are restructuring lung cancer screening.
Thyroid disease in pregnancy management formalises around structured monitoring
Pregnancy-specific TSH targets, structured pre-conception thyroid management, and integrated maternal-fetal medicine and endocrinology pathways are reshaping thyroid disease in pregnancy.
IgG4-related disease enters real prescribing territory
Inebilizumab IgG4-RD pivotal data, rituximab maintenance protocols, and structured diagnostic pathways are reshaping IgG4-related disease management.
Frontotemporal dementia therapy programs reach late-stage trials
Progranulin-replacement therapy in GRN-mutation FTD, ASO programs in C9orf72 FTD-ALS, and tau-targeted programs are emerging in a previously bare category.
Dementia with Lewy bodies care formalises around alpha-synuclein-aware management
Cholinesterase inhibitor optimisation, antipsychotic-avoidance protocols, RBD recognition pathways, and emerging alpha-synuclein-targeted programs are reshaping DLB care.
Proliferative diabetic retinopathy therapy reshapes around anti-VEGF
Anti-VEGF first-line for proliferative diabetic retinopathy, panretinal photocoagulation as alternative or combination, and emerging mechanism programs are reshaping PDR management.
Spinal muscular atrophy long-term outcome data reshapes treatment expectations
Multi-year outcome data across nusinersen, onasemnogene abeparvovec, and risdiplam plus emerging combination strategies are clarifying long-term SMA treatment expectations.
Keratoconus therapy widens past corneal cross-linking
Corneal cross-linking adoption, intracorneal ring segment maturity, and emerging custom cross-linking and topography-guided treatment are reshaping keratoconus management.
Refractory chronic cough acquires a first targeted mechanism class
P2X3 antagonist class entry plus follow-on novel mechanism programs are establishing chronic cough as a real prescribing category.
Stroke prevention restructures across atrial fibrillation, lipids, and acute window
Factor XIa inhibitors entering late-stage trials, expanded thrombectomy windows, and tenecteplase displacement of alteplase are restructuring stroke prevention and acute care.
Postmenopausal osteoporosis therapy reshapes around anabolic-first sequencing
Romosozumab maturity, ongoing teriparatide and abaloparatide use, and follow-on anabolic programs are restructuring postmenopausal osteoporosis sequencing.
Pre-eclampsia prediction and prevention infrastructure matures
sFlt-1 to PlGF ratio testing in suspected pre-eclampsia and aspirin-prophylaxis pathway adoption are reshaping pre-eclampsia care.
MASH therapy class establishes after resmetirom approval
Resmetirom commercial uptake plus follow-on FGF21 analogues, GLP-1 plus glucagon, and PPAR-pan agonists are establishing a real metabolic-dysfunction-associated steatohepatitis prescribing class.
Hidradenitis suppurativa biologic options widen past TNF
IL-17-class secukinumab and bimekizumab approvals plus emerging novel mechanism programs are restructuring hidradenitis suppurativa management.
Diabetic peripheral neuropathy therapy advances after a long quiet period
Capsaicin patch maturity, novel sodium channel modulators, and emerging disease-modifying programs are reshaping diabetic peripheral neuropathy management.
Sjogren disease therapy enters real prescribing territory
First positive pivotal readouts in Sjogren disease are establishing a category that has had no specific systemic therapy for decades.
Inherited retinal disease gene therapy widens past RPE65
X-linked retinitis pigmentosa, choroideremia, and additional inherited retinal disease gene therapy programs are reading out.
Tardive dyskinesia therapy access patterns
Approved VMAT2 inhibitor therapy for tardive dyskinesia continues to expand but underdiagnosis remains the primary gap.
Vaping-associated lung disease as a recognised category
EVALI (e-cigarette and vaping-associated lung injury) and chronic bronchiolitis from vaping are now recognised pulmonology diagnostic categories.
Concussion and TBI biomarker tools mature
Plasma GFAP and UCH-L1 assays for concussion and traumatic brain injury are establishing routine emergency-department use.
Non-CF bronchiectasis enters the therapy frontier
Late-stage programs targeting non-CF bronchiectasis are creating the first specific therapy category for this long-overlooked condition.
FDA approves leucovorin calcium as first treatment for CFD-FOLR1
The FDA has approved expanded use of Wellcovorin (leucovorin calcium) tablets for cerebral folate deficiency caused by confirmed FOLR1 gene variants, marking the first approved treatment for this rare neurological condition in adults and children.
Plasma biomarker testing is displacing amyloid PET as the screening modality for anti-amyloid eligibility
Three FDA-cleared blood tests for amyloid pathology are now in routine use at major Alzheimer's centres, materially reducing the amyloid-PET demand that constrained early lecanemab uptake.
ATTR amyloidosis silencer therapy uptake is transforming a previously underdiagnosed condition
Transthyretin amyloid cardiomyopathy (ATTR-CM) was historically underdiagnosed and undertreated. Approved silencer therapies (siRNA, ASO) and TTR stabilisers have moved the field rapidly, and the diagnostic-pathway access remains the rate-limit on commercial uptake.
Parkinson's biomarker work is converging on the alpha-synuclein seed-amplification assay
The alpha-synuclein seed-amplification assay (alpha-syn-SAA) has emerged as the leading biomarker for Parkinson's disease and adjacent synucleinopathies. The assay's sensitivity, specificity, and the implications for both diagnosis and trial-enrolment are material.
UK newborn screening expansion proposal would change the rare-disease patient-finding model
The UK National Screening Committee's proposed expansion of the newborn blood-spot screening panel to additional rare conditions has implications for diagnosis timing, treatment-eligible patient identification, and commercial planning for orphan therapies in those conditions.
Newborn screening expansion is uneven across US states
RUSP additions tell only half the story - state-level implementation timelines stretch the diagnostic-to-treatment gap, and the variation has consequences for treatment-eligibility windows in time-sensitive rare disease.
Tau PET reimbursement is the next diagnostic access question
Amyloid PET has settled into routine coverage and availability. Tau PET - which will be the confirmatory pathway for tau-directed therapies - is sited at meaningfully fewer centers and reimbursed unevenly. The access conversation that defined anti-amyloid rollout is about to repeat, one mechanism later.
Co-pathology recognition is reshaping how Alzheimer's diagnosis is being read
LATE, vascular contribution, and Lewy-body co-pathology are now routinely on the differential when a patient with cognitive symptoms tests amyloid-positive. The clinical question is increasingly "what mix" rather than "is it Alzheimer's."
Plasma-biomarker rollout is concentrated at academic centers
Adoption of plasma p-tau217 testing remains concentrated at academic medical centers and large specialty practices, with community uptake meaningfully behind.
Blood-based biomarkers move from research to clinical workflow
Plasma p-tau217 assays are being adopted as a triage step before PET or CSF confirmation in specialty memory clinics.
Snapshots
18Cardiac amyloidosis therapy reference (2026)
Reference snapshot of cardiac amyloidosis therapy across AL and ATTR types.
Retinal vein occlusion therapy reference (2026)
Reference snapshot of RVO therapy across acute and ongoing macular edema management plus systemic care integration.
Dementia subtype therapy reference (2026)
Reference snapshot of dementia therapy across Alzheimer's, dementia with Lewy bodies, vascular, and frontotemporal subtypes.
Allergic bronchopulmonary aspergillosis therapy reference (2026)
Reference snapshot of ABPA therapy across acute, recurrent, and emerging biologic-integrated tiers.
Chronic inflammatory demyelinating polyneuropathy therapy reference (2026)
Reference snapshot of CIDP therapy across induction, maintenance, and emerging biologic tiers.
Allergic conjunctivitis therapy reference (2026)
Reference snapshot of allergic conjunctivitis therapy across mild seasonal, persistent, severe, and emerging tiers.
Phenylketonuria therapy reference (2026)
Reference snapshot of PKU therapy across newborn-screened, paediatric, and adult populations.
Autism spectrum disorder care reference (2026)
Reference snapshot of ASD care across behavioural therapy, educational support, pharmacotherapy for associated symptoms, and emerging core-symptom programs.
Spinal muscular atrophy therapy reference (2026)
Reference snapshot of SMA therapy options across newborn-screened, symptomatic infant, child, and adult populations.
Obstructive sleep apnea therapy reference (2026)
Reference snapshot of OSA therapy across positive airway pressure, oral appliances, surgery, neurostimulation, and emerging pharmacotherapy.
Sjogren disease therapy reference (2026)
Reference snapshot of Sjogren disease therapy across symptomatic, off-label, and emerging mechanism-targeted tiers.
Hereditary angioedema therapy reference (2026)
Reference snapshot of HAE prophylaxis and on-demand therapy options.
Refractory chronic cough therapy reference (2026)
Reference snapshot of refractory chronic cough therapy across symptomatic, off-label, and emerging targeted tiers.
Hepatocellular carcinoma therapy reference (2026)
Reference snapshot of HCC therapy across early, intermediate, and advanced disease.
Type 1 diabetes therapy reference (2026)
Reference snapshot of T1D therapy across insulin delivery, disease modification, and beta-cell replacement.
Postmenopausal osteoporosis therapy reference (2026)
Reference snapshot of postmenopausal osteoporosis therapy across anti-resorptive and anabolic tiers.
ATTR cardiomyopathy treatment landscape, 2026 mid-year reference
Reference layout of the ATTR cardiomyopathy treatment landscape as of mid-2026: TTR stabilisers, TTR silencers, the diagnostic-pathway infrastructure, and the live commercial questions including diagnostic-pathway expansion and combination therapy.
Alzheimer's diagnostic pathways, as of Q2 2026
Plasma biomarkers are entering routine workflows alongside established CSF and PET options, with confirmatory imaging still standard before disease-modifying therapy.
Explained
45What is AL amyloidosis?
Plain-language primer on AL amyloidosis, why early diagnosis matters so much, and how modern therapy works.
What is hyperemesis gravidarum?
Plain-language primer on hyperemesis gravidarum, why it is more than morning sickness, and what comprehensive care can offer.
What is systemic sclerosis?
Plain-language primer on systemic sclerosis (scleroderma), why it affects multiple organ systems, and what modern therapy can offer.
What is allergic bronchopulmonary aspergillosis?
Plain-language primer on ABPA, why it is different from typical asthma, and how modern therapy works.
What is achondroplasia?
Plain-language primer on achondroplasia, why it is the most common skeletal dysplasia, and what the new therapy options can offer.
What is thyroid cancer?
Plain-language primer on thyroid cancer, why molecular profile now matters, and how modern therapy works.
What is dementia with Lewy bodies?
Plain-language primer on dementia with Lewy bodies, why it is different from Alzheimer's, and how modern care works.
What is retinal vein occlusion?
Plain-language primer on retinal vein occlusion, why it is a vascular event, and what modern therapy can offer.
Autism spectrum disorder care explained
Plain-language primer on ASD care, why it is multidisciplinary, and what is changing in pharmacotherapy.
PCSK9 inhibitors and modern lipid therapy explained
Plain-language primer on PCSK9 and the modern range of lipid-lowering options.
What is ANCA-associated vasculitis?
Plain-language primer on ANCA-associated vasculitis, why it can affect multiple organs, and how modern therapy works.
What is cholangiocarcinoma?
Plain-language primer on cholangiocarcinoma, why molecular profiling now matters so much, and how modern therapy works.
What is overactive bladder?
Plain-language primer on overactive bladder, why it is common, and what the modern therapy options can offer.
What is chronic inflammatory demyelinating polyneuropathy?
Plain-language primer on CIDP, why immune therapy is the foundation, and what the modern options can offer.
What is phenylketonuria?
Plain-language primer on phenylketonuria, why early diagnosis matters so much, and what the modern therapy options can offer.
What is allergic conjunctivitis?
Plain-language primer on allergic conjunctivitis, the different types, and what the modern range of therapy can offer.
Severe COPD and biologic therapy explained
Plain-language primer on severe COPD, why biologic therapy is now an option for some patients, and what the modern combined approach can offer.
What is lupus nephritis?
Plain-language primer on lupus nephritis, why kidney involvement is a turning point in lupus, and how modern therapy works.
What are vasomotor symptoms?
Plain-language primer on hot flashes and night sweats, why they happen, and what the modern therapy options can offer.
What is spinal muscular atrophy?
Plain-language primer on SMA, why genetics drives the disease, and how the modern therapy options work.
What is glioblastoma?
Plain-language primer on glioblastoma, why it has been so hard to treat, and what is changing in glioma therapy.
What is obstructive sleep apnea?
Plain-language primer on obstructive sleep apnea, why CPAP has been the standard, and what is changing in OSA therapy.
What is spasticity?
Plain-language primer on spasticity, why it happens after neurological injury, and what the therapy options can offer.
What is heart failure?
Plain-language primer on heart failure, why the modern therapy is built around four foundational classes, and what is changing.
Acute ischemic stroke explained
Plain-language primer on acute ischemic stroke, why time matters, and how modern therapy works.
What is hepatocellular carcinoma?
Plain-language primer on hepatocellular carcinoma, why it usually develops on a background of liver disease, and how the modern therapy options compare.
What is type 1 diabetes?
Plain-language primer on type 1 diabetes, what is changing, and how modern therapy works.
What is obsessive-compulsive disorder?
Plain-language primer on OCD, why it has multiple subtypes, and what therapy can offer.
What is thyroid eye disease?
Plain-language primer on thyroid eye disease, why it is linked to thyroid problems, and what modern therapy can offer.
What is Sjogren disease?
Plain-language primer on Sjogren disease, why it has been hard to treat, and what is changing.
What is hereditary angioedema?
Plain-language primer on hereditary angioedema, why it is different from allergy, and how modern therapy works.
Postmenopausal osteoporosis explained
Plain-language primer on postmenopausal osteoporosis, why bone changes after menopause, and how modern therapy works.
What is refractory chronic cough?
Plain-language primer on refractory chronic cough, why it is a distinct condition, and what is changing in therapy.
What ARIA is, and why it gates how anti-amyloid antibodies can be used
ARIA - amyloid-related imaging abnormalities - is the side effect that defines the operational and clinical experience of being on lecanemab or donanemab. Understanding what it is, who it affects more, and why it requires MRI surveillance is essential context for any conversation about anti-amyloid treatment.
How inherited retinal dystrophy gene therapy is moving beyond RPE65
The voretigene neparvovec approval for RPE65-mediated inherited retinal dystrophy validated AAV-based gene therapy in ophthalmology. The pipeline has moved substantially beyond RPE65 to address adjacent inherited retinal dystrophy genotypes, with implications for diagnostic infrastructure, surgical delivery, and commercial planning.
Preeclampsia screening: where biomarker-based pathways are converting
Preeclampsia screening has moved from clinical-risk-factor-driven to biomarker-augmented pathways in several markets. The methodology, the access frame, and the implications for both maternal outcomes and commercial planning around adjacent assets are worth understanding.
ctDNA-guided treatment decisions are entering routine oncology care
Circulating tumour DNA testing has moved from research-grade tool to a routine decision aid in several oncology indications. The pathways from blood draw to clinical decision are now described well enough to plan around, even though reimbursement remains uneven.
What are CSF biomarkers, and how do they fit alongside plasma and PET?
CSF biomarkers - measured in cerebrospinal fluid obtained by lumbar puncture - are well-validated tests for Alzheimer's pathology that pre-date both plasma biomarkers and broad amyloid PET access. They remain in routine clinical use, particularly where PET is not available and where the diagnostic question is complex enough to warrant the procedural friction.
What is tau PET, and how is it different from amyloid PET?
Tau PET is a brain scan that shows the build-up of tau, the second protein associated with Alzheimer's disease. The image is closer to the clinical picture than amyloid is - tau accumulation tracks more directly with where and how a person is currently impaired. The trade-off is that tau PET is harder to access than amyloid PET, and the access gap is now a rate-limiter on multiple fronts.
What is mild cognitive impairment, and how is it different from "early Alzheimer's"?
Mild cognitive impairment (MCI) is a diagnostic category, not a disease. It describes cognitive decline that is more than expected for age but not severe enough to count as dementia. When that decline is caused by underlying Alzheimer's pathology, the term you will increasingly hear in clinical-trial and treatment settings is "early Alzheimer's disease" - which means MCI due to Alzheimer's plus mild dementia due to Alzheimer's, taken together.
What is CDR-SB, and what does a small change on it actually mean?
CDR-SB is the Clinical Dementia Rating - Sum of Boxes, the cognitive and functional scale used as the primary endpoint in most late-stage Alzheimer's trials. It is a six-box, 0–18 scale, scored by a clinician from a structured interview with the patient and a caregiver.
What is co-pathology in dementia, and why does it matter?
Co-pathology means more than one disease process is contributing to someone's symptoms at the same time. In older patients with cognitive symptoms, mixed pathology is the rule, not the exception.
What is amyloid PET, and when is it used?
Amyloid PET is a brain scan that shows whether the amyloid protein associated with Alzheimer's disease has built up in the brain. It is used to confirm or rule out Alzheimer's pathology when the diagnosis matters.
What are plasma biomarkers in Alzheimer's disease?
Plasma biomarkers are blood tests that look for proteins associated with Alzheimer's disease pathology. The most clinically useful one in 2026 is plasma p-tau217.
Why APOE4 matters in Alzheimer's disease and treatment
APOE4 is both the strongest common genetic risk factor for late-onset Alzheimer's and a meaningful modifier of treatment safety - which is why genotyping is now part of the workup.