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AstraZeneca PLC

Drugmaker with rare disease work · based in Cambridge, United Kingdom · founded 1999 · ticker AZN · astrazeneca.com

AstraZeneca PLC works on 50 rare diseases we track, including Acute Myeloid Leukemia, Adrenocortical Carcinoma and 48 more. It has 18 active clinical trials, and 14 are looking for volunteers now. It also has 8 FDA-approved rare disease drugs.

The FDA is due to decide on Efzimfotase alfa (ALXN1850) for Hypophosphatasia in the first half of 2027.

Email me the day the FDA decidesAll upcoming rare disease FDA decisions

AstraZeneca was formed in 1999 by the merger of Astra and Zeneca. It entered rare disease with Alexion, a deal announced at $39 billion and completed on July 21, 2021, and Alexion now runs as AstraZeneca's rare disease unit. Alexion's medicines, including Soliris, Ultomiris and Voydeya, are covered on the Alexion Pharmaceuticals page.

AstraZeneca PLC pipeline

Each row lists the diseases AstraZeneca PLC is studying at that stage, from the ones closest to approval down to the earliest work.

Approved
Drugs the FDA has approved.
At the FDA
Waiting on an FDA decision.

Efzimfotase alfa (ALXN1850) for Hypophosphatasia, with a decision due in the first half of 2027.

Phase 3
Large studies, usually the last step before a company asks the FDA for approval.
Phase 2
Mid-size studies that check whether a drug works and at what dose.

B-cell Non-Hodgkin Lymphoma 2 trials recruiting · Systemic Sclerosis recruiting · Cholangiocarcinoma recruiting · Multiple Myeloma recruiting · Mesothelioma recruiting · Recurrent Moderate-Severe Chronic Graft Versus Host Disease · Fibrodysplasia Ossificans Progressiva · Asthma

Phase 1
First studies in people, mostly checking safety.
Other studies
Studies with no phase listed, such as registries and natural history studies.

AstraZeneca PLC clinical trials

These are AstraZeneca PLC trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

14 trials looking for volunteers now

Looking for volunteersRecently updatedNCT07654855

Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations

Tests None ( observational study )

For Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy and Transthyretin Amyloidosis With Polyneuropathy

The aim of this observational cohort study is to characterise use of eplontersen in patients with prior liver transplant or with pre-existing severe hepatic impairment, as well as to assess long-term safety among all new users of eplontersen; all are areas of missing information Primary objectives are: 1. To describe demographic and clinical characteristics of patients at eplontersen initiation, including the prevalence of prior liver transplant (overall and by reason for liver transplant), and the prevalence of severe hepatic impairment; and to describe patients in these subgroups (prior liver transplant, severe hepatic impairment). 2. To describe long-term safety in patients who initiate eplontersen treatment, including onset of new clinical events, abnormal laboratory values and serious adverse events.

Ages 18 Years+6 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07691970

Rollover Study for Participants Who Have Completed a Previous Clinical Study With Anifrolumab (Saphnelo) and Clinically Benefited From Continued Treatment

Tests Anifrolumab, Anifrolumab, Anifrolumab

For Systemic Sclerosis Cutaneous Lupus E Myopathies Systemic Lupus Erythematosus

The purpose of this study is to provide continued access to anifrolumab to eligible participants who are continuing to derive clinical benefit from treatment at the end of a parent clinical study, as assessed by the Investigator, while monitoring safety and tolerability. Study details include: * Participants can transition in ROSY-S once they complete their participation in the parent study and should have received at least one dose of anifrolumab in the parent study. Participants with a treatment gap from parent studies and/or who are on compassionate use are eligible to rollover to ROSY-S study. * The earliest that the Enrolment Visit may occur is at the time of the last visit of the parent study (ie, completion of the parent study's final safety follow-up visit). All required assessments from the safety follow-up visit of the parent study must be completed before the Enrolment Visit of ROSY-S study. * The participants will continue to receive anifrolumab the same way they received it in the parent trial * A participant is considered to have completed the study when they meet one of the discontinuation criteria. * Safety will be monitored throughout the study. Both serious and nonserious AEs will be collected up to 12 weeks after the last dose of anifrolumab and will be reported as part of the primary safety endpoint.

Ages 5 Years - 80 Years166 locations
View full study
Looking for volunteersPhase 1/2Recently updatedNCT07295847

A Study of AZD0120 in Autoimmune Diseases

Tests AZD0120, SOC

For Systemic Sclerosis, Idiopathic Inflammatory Myopathies and Rheumatoid Arthritis

This trial is a Phase 1b/2, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and tolerability in adult participants with systemic sclerosis (SSc), idiopathic inflammatory myopathies (IIM), or difficult-to-treat rheumatoid arthritis (D2T RA).

Ages 18 Years - 75 Years27 locations
View full study
Looking for volunteersPhase 1/2Recently updatedNCT07123454

A Phase I/II Study of AZD4512 Monotherapy or in Combination With Anticancer Agents in Participants With Relapsed/Refractory B-cell Non-Hodgkin Lymphoma

Tests AZD4512, Surovatamig

For B-cell Non-Hodgkin Lymphoma

This is a phase I/II open-label, global multicenter study to evaluate the safety and efficacy of AZD4512 monotherapy or in combination with other anticancer agent(s), in participants with relapsed/refractory B-cell non-Hodgkin lymphoma (B-NHL).

Ages 18 Years+23 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT06512883

A Trial to Investigate Benralizumab in Children With Eosinophilic Diseases

Tests Benralizumab

For Eosinophilic Granulomatosis With Polyangiitis (EGPA) and Hypereosinophilia Syndrome (HES)

The main purpose of study is to assess the safety, tolerability, pharmacokinetics (PK), and efficacy of benralizumab.

Ages 6 Years - 17 Years15 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT06440993

Durvalumab + Intraductal Radiofrequency Ablation (ID-RFA) in Extra- and Intrahepatic Cholangiocarcinoma

Tests Gemcitabine, Cisplatin, Durvalumab, ID-RFA

For Extrahepatic Cholangiocarcinoma and Unresectable Perihilar or Ductal CCA

The present clinical trial is a prospective, investigator-initiated, single-arm, open-label, multicenter phase II trial. Patients with unresectable perihilar and/or ductal or intrahepatic CCA with indication for bile duct stenting and palliative systemic therapy as determined by the local multidisciplinary team (MDT), who already resolved cholestasis due to RFA + Stent will be enrolled. We hypothesize that in patients with extra- and intrahepatic cholangiocarcinoma, the use of a combination radiofrequency ablation followed by systemic treatment with chemotherapy plus durvalumab might further increase the anti-tumor activity.

Ages 18 Years+12 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT06526793

Surovatamig (AZD0486) as Monotherapy in Participants With Relapsed/Refractory (R/R) B-cell NHL

Tests Surovatamig

For B-cell Non-Hodgkin Lymphoma, Follicular Lymphoma (FL) and Large B-Cell Lymphoma (LBCL)

This is a Phase 2 global, multi-center, open-label study to assess the efficacy, safety and tolerability of surovatamig (AZD0486) monotherapy in adult participants with relapsed/refractory B-cell non-Hodgkin lymphoma (NHL) who have received at least two prior lines of therapies. The study has 2 Modules: Module 1 for FL and Module 2 for LBCL.

Ages 18 Years+94 locations
View full study
Looking for volunteersRecently updatedNCT07247279

Epidemiological Study of Treatment Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis in Russia

For Rare Diseases and Generalized Myasthenia Gravis (gMG)

This is a multicenter, non-interventional, retrospective-prospective, single-arm observational study designed to describe real-world treatment approaches and clinical outcomes among adults with acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) in routine clinical practice in Russia.

Ages 18 Years+7 locations
View full study
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FDA-approved drugs from AstraZeneca PLC

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Baxfendy baxdrostat

For Adrenocortical Carcinoma. Approved May 15, 2026. Taken by mouth.

Calquence acalabrutinib

For Mantle Cell Lymphoma. Approved Oct 31, 2017. Taken by mouth.

Crestor rosuvastatin

For Homozygous Familial Hypercholesterolemia. Approved Aug 12, 2003. Taken by mouth.

Fasenra benralizumab

For Eosinophilic Granulomatosis with Polyangiitis. Approved Nov 14, 2017. Taken by subcutaneous route.

Imfinzi durvalumab

For Cholangiocarcinoma. Approved May 1, 2017.

Koselugo selumetinib

For Neurofibromatosis Type 1. Approved Apr 10, 2020. Taken by mouth.

Saphnelo anifrolumab-fnia

For Lupus Nephritis. Approved Jul 30, 2021. Taken by intravenous, subcutaneous route.

Wainua eplontersen

Approved Dec 21, 2023. Taken by subcutaneous route.

More about AstraZeneca PLC

AstraZeneca's own rare disease medicines include Koselugo (selumetinib) for plexiform neurofibromas in people with NF1 aged 1 and older, Wainua (eplontersen) for the nerve damage of hereditary ATTR amyloidosis in adults, and Fasenra (benralizumab) for adults with EGPA. Ultomiris received accelerated approval for IgA nephropathy, announced on October 21, 2026, and the FDA is reviewing efzimfotase alfa for hypophosphatasia and gefurulimab for generalized myasthenia gravis.

Where AstraZeneca PLC trials take place

The trials above run at 771 study sites in 41 countries. Tap a country to see its states, cities and hospitals.

United States
300▼
China
100▼
Germany
60▼
Spain
30▼
United Kingdom
25▼
Italy
22▼
Algeria
21▼
Taiwan
19▼
Canada
18▼
Australia
18▼
Japan
18▼
Brazil
17▼

Rare diseases AstraZeneca PLC works on

Each disease page lists every open trial, not only AstraZeneca PLC's, plus approved treatments.

Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a fast-growing cancer of the bone marrow in which immature white blood cells called blasts crowd out normal blood cells, causing fatigue, infections and bleeding...

How common: The National Cancer Institute's SEER program estimates 22,720 new US cases and 11,500 deaths in 2026, about 1.1% of new cancers, and 83,311 people were living with AML in 2023

Adrenocortical Carcinoma

Adrenocortical carcinoma is a rare, aggressive cancer of the adrenal cortex, the outer layer of the adrenal glands that sit on top of each kidney.

How common: About 1-2 per million people per year; approximately 600 new cases per year in the U.S

ATTR Amyloidosis (Transthyretin Amyloidosis)

ATTR amyloidosis is a rare progressive disease where abnormal transthyretin protein accumulates as amyloid deposits in the heart, nerves, and other organs.

How common: Approximately 5,000 to 7,000 new cases diagnosed annually in the U.S

Atypical Hemolytic Uremic Syndrome

Atypical Hemolytic Uremic Syndrome (aHUS) is a rare kidney disease caused by uncontrolled activation of the complement system, a part of the immune system that normally helps fight infection.

How common: Approximately 1-2 cases per million people per year; rare form of HUS accounting for 5-10% of all HUS cases

Bullous Pemphigoid

Bullous pemphigoid is an autoimmune blistering disorder caused by autoantibodies against basement membrane proteins, particularly BP180 and BP230.

How common: Approximately 2-23 per million people per year; incidence increases sharply with age

Cholangiocarcinoma

Cholangiocarcinoma is a rare and aggressive cancer that forms in the bile ducts, the thin tubes that carry digestive fluid (bile) from the liver to the small intestine.

How common: About 8,000 new cases per year in the U.S.; rising incidence worldwide

Chronic Graft-versus-Host Disease

Chronic graft-versus-host disease is an immune-mediated complication after allogeneic stem cell or bone marrow transplant, in which donor immune cells attack the recipient's tissues.

How common: Affects 30-70% of patients who receive allogeneic hematopoietic stem cell transplant; about 14,000 Americans were living with chronic GVHD in 2016, according to a claims study funded in part by Kadmon (Bachier et al., Transplantation and Cellular Therapy, 2021)

Cushing Disease

Cushing Disease is a rare endocrine disorder caused by an ACTH-secreting pituitary adenoma that leads to excessive cortisol production.

How common: Estimated 2-8 new cases per million people per year, though recent population studies suggest the true incidence may be higher

Cystic Fibrosis

Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport.

How common: Nearly 40,000 children and adults in the U.S.; 1 in 2,500 to 3,500 births among Caucasians

Dermatomyositis

Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints.

How common: About 9.6 new cases per million people a year, according to NORD; juvenile-onset incidence about 0.4 cases per 100,000 children

Diffuse Intrinsic Pontine Glioma

Diffuse intrinsic pontine glioma (DIPG) is an aggressive childhood brain tumor that grows in the pons, the part of the brainstem controlling breathing, swallowing, and movement.

How common: About 300 children diagnosed in the U.S. each year, most often between ages 5 and 10

Dilated Cardiomyopathy

Dilated cardiomyopathy is a condition where the heart's main pumping chamber, the left ventricle, becomes enlarged and weakened, reducing the heart's ability to pump blood efficiently.

How common: Approximately 1 in 250 to 1 in 500 people; accounts for about 30-40% of heart failure cases

Eosinophilic Esophagitis

Eosinophilic Esophagitis is a rare allergic/immune-mediated inflammatory disease of the esophagus characterized by eosinophil infiltration causing progressive narrowing and dysfunction.

How common: About 142.5 per 100,000 people in the United States, about 1 in 700 or an estimated 472,380 people, in a 2025 insurance claims study (Thel et al., Clin Gastroenterol Hepatol 2025)

Eosinophilic Granulomatosis with Polyangiitis

Eosinophilic granulomatosis with polyangiitis (EGPA, formerly Churg-Strauss syndrome) is a systemic necrotizing vasculitis characterized by asthma, blood eosinophilia, and inflammation of small- and...

How common: 10 to 14 cases per million people; approximately 3,000 to 4,500 individuals in the U.S

Essential Thrombocythemia

Essential Thrombocythemia is a myeloproliferative neoplasm characterized by overproduction of platelets by bone marrow, resulting in very high platelet counts.

How common: About 1 new case per 100,000 people each year, with an estimated 30 to 60 per 100,000 living with it (NORD); average age at diagnosis is in the mid-fifties

Fibrodysplasia Ossificans Progressiva

Fibrodysplasia Ossificans Progressiva (FOP) is an extremely rare genetic disorder characterized by progressive heterotopic ossification, where soft tissues (muscles, tendons, ligaments) gradually...

How common: Extremely rare, about 1 in 1 million to 1 in 2 million people worldwide

Focal Segmental Glomerulosclerosis

Focal Segmental Glomerulosclerosis (FSGS) is a kidney disease characterized by scarring of some glomeruli, the tiny filtering units that remove waste from the blood.

How common: Approximately 7% of all kidney biopsies in the United States show FSGS; incidence is 0.2-1.7 cases per 100,000 person-years

Gastrointestinal Stromal Tumor

Gastrointestinal stromal tumors (GISTs) are the most common mesenchymal tumors of the gastrointestinal tract, arising from the interstitial cells of Cajal or their precursors.

How common: About 6,000 new cases per year in the U.S. (American Cancer Society); true incidence may be higher as small GISTs are often incidentally discovered

Gastroparesis

Gastroparesis is a condition in which the stomach empties food too slowly even though nothing is blocking it, causing early fullness, nausea, vomiting, bloating and upper belly pain.

How common: In a Minnesota population study cited by the NIDDK, about 10 men and 40 women per 100,000 had diagnosed gastroparesis

Granulomatosis with Polyangiitis

Granulomatosis with Polyangiitis is a rare autoimmune disease causing inflammation of small and medium blood vessels in the lungs, kidneys, and upper respiratory tract.

How common: An estimated 3 in 100,000 people in the United States, according to MedlinePlus Genetics

Hereditary Diffuse Gastric Cancer

Hereditary diffuse gastric cancer is an autosomal dominant cancer predisposition syndrome caused by germline CDH1 mutations.

How common: Estimated at 1 in 1,000 gastric cancer cases; ~1 in 100,000 in general population

Hodgkin Lymphoma

Hodgkin lymphoma is a cancer of the lymphatic system characterized by the presence of abnormal Reed-Sternberg cells.

How common: About 8,920 new cases expected in the U.S. in 2026 (American Cancer Society); approximately 2.6 per 100,000 people

Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia is a severe genetic disorder causing extremely high cholesterol levels from birth, leading to early heart disease and cholesterol deposits in tendons and...

How common: Approximately 1 in 250,000 to 1 in 360,000 people globally

Hypophosphatasia

Hypophosphatasia (HPP) is a rare inherited disorder in which the body makes too little of an enzyme called alkaline phosphatase, so bones and teeth do not harden properly.

How common: Severe HPP that shows up at birth or in infancy affects about 1 in 100,000 live births in Canada, the figure NORD and Soft Bones both use for North America

Idiopathic Pulmonary Fibrosis

Idiopathic pulmonary fibrosis is a chronic, progressive lung disease characterized by scarring (fibrosis) of lung tissue.

How common: About 100,000 people in the U.S., with 30,000 to 40,000 new cases a year (MedlinePlus Genetics)

Immune Thrombocytopenia

Immune thrombocytopenia (ITP) is a rare autoimmune disorder in which the immune system destroys platelets, the blood cells that stop bleeding, and slows their production.

How common: Estimated 9 to 20 per 100,000 people; children often have a temporary form that resolves within months

Lupus Nephritis

Lupus nephritis is kidney inflammation caused by systemic lupus erythematosus (SLE), in which the immune system attacks the kidneys' filters and, without treatment, can lead to kidney failure.

How common: About 21 per 100,000 people in a Minnesota population study in 2015 (Lupus Midwest Network, 2022)

Mantle Cell Lymphoma

Mantle cell lymphoma is a rare type of non-Hodgkin lymphoma that starts in B cells, a kind of white blood cell.

How common: About 1 in 200,000 people (Cleveland Clinic)

Medullary Thyroid Cancer

Medullary thyroid cancer (MTC) is a rare neuroendocrine tumor arising from the parafollicular C cells of the thyroid gland, accounting for 2-4% of all thyroid cancers.

How common: Approximately 1,000-1,200 new cases per year in the U.S.; 2-4% of all thyroid cancers

Membranous Nephropathy

Membranous nephropathy is a rare autoimmune kidney disease where the body's own antibodies attack proteins in the kidney's filtering units (glomeruli).

How common: About 12 new cases per million people each year in the U.S.; roughly 40,000-50,000 Americans living with the disease

Merkel Cell Carcinoma

Merkel cell carcinoma is a rare, aggressive skin cancer that arises from Merkel cells, specialized touch receptors in the skin.

How common: About 3,000 new cases per year in the U.S.; incidence has tripled over the past 2 decades

Mesothelioma

Mesothelioma is a rare, aggressive cancer that develops in the mesothelium, the thin layer of tissue covering most internal organs.

How common: About 3,000 new cases per year in the U.S

Multiple Myeloma

Multiple myeloma is a cancer of plasma cells, the white blood cells that make antibodies.

How common: About 36,000 new US diagnoses expected in 2026 and roughly 203,000 people living with it, which puts myeloma right at the edge of the US rare disease threshold; it is 1.7% of all new cancers (NCI SEER)

Multiple Sclerosis

Multiple sclerosis is a chronic autoimmune disease where the immune system attacks the protective myelin sheath surrounding nerve fibers in the brain and spinal cord.

How common: Approximately 1 million people in the U.S.; about 1 in 300

Multiple System Atrophy

Multiple system atrophy is a rare, progressive neurodegenerative disorder characterized by dysfunction of the autonomic nervous system and movement disorders.

How common: 2-5 per 100,000 people

Myasthenia Gravis

Myasthenia gravis is a chronic autoimmune neuromuscular disorder causing muscle weakness and rapid fatigue.

How common: 1 in 5,000 people

Myelodysplastic Syndromes

Myelodysplastic syndromes are a group of blood cancers in which the bone marrow fails to produce enough healthy blood cells.

How common: 10,000 to 15,000 new cases annually in the U.S.; estimated 60,000 to 170,000 Americans living with MDS

Myelofibrosis

Myelofibrosis is a myeloproliferative neoplasm in which bone marrow develops scarring (fibrosis), leading to anemia, enlarged spleen, and abnormal blood cell counts.

How common: Approximately 0.5-1.5 cases per 100,000 people; median age of diagnosis is 65 years

Neurofibromatosis Type 1

Neurofibromatosis type 1 is a genetic disorder caused by mutations in the NF1 gene on chromosome 17, which encodes neurofibromin, a tumor suppressor protein.

How common: 1 in 2,500 to 3,500 people in the U.S., making it one of the most common genetic conditions

Neuromyelitis Optica

Neuromyelitis Optica (NMO) is a rare inflammatory autoimmune disorder of the central nervous system characterized by recurrent attacks of optic neuritis (vision loss) and acute myelitis (spinal cord...

How common: About 1 to 2 per 100,000 people worldwide, according to MedlinePlus Genetics

Paraganglioma

Paragangliomas are rare neuroendocrine tumors arising from chromaffin tissue outside the adrenal medulla.

How common: 1 in 300,000 to 1,000,000; ~30% are hereditary

Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria is a rare, life-threatening blood disorder where complement-mediated destruction of red blood cells occurs due to acquired mutations in the PIGA gene.

How common: Estimated at 1 to 5 per million people (MedlinePlus Genetics); NORD gives 0.5 to 1.5 per million

Polycystic Kidney Disease

Polycystic Kidney Disease is a genetic disorder characterized by the growth of thousands of cysts in the kidneys, gradually replacing normal kidney tissue and leading to kidney failure.

How common: Autosomal dominant PKD affects about 1 in 400 to 1,000 people worldwide and the recessive form about 1 in 20,000 children; about 500,000 people in the United States have PKD (NIDDK)

Primary Central Nervous System Lymphoma

Primary central nervous system lymphoma (PCNSL) is a fast-growing lymphoma that starts in the brain, spinal cord, eyes or the membranes around them and usually stays there.

How common: About 5 new cases per million people a year in the US (Ono Pharmaceutical and Deciphera); more than half of patients are 60 or older

Pulmonary Arterial Hypertension

Pulmonary arterial hypertension is a rare disease characterized by abnormally high blood pressure in the pulmonary arteries, causing progressive heart and lung damage.

How common: About 26 to 52 per million people in a Scottish national study, with about 7 new cases per million people each year (Peacock et al., European Respiratory Journal, 2007)

Sickle Cell Disease

Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion.

How common: About 100,000 Americans with SCD; about 1 in 365 Black or African American births and 1 in 16,300 Hispanic American births

Sjögren's Syndrome

Sjögren's syndrome is a chronic autoimmune disorder in which the immune system attacks the moisture-producing exocrine glands, primarily the salivary and lacrimal (tear) glands.

How common: Estimated 1 to 4 million Americans (including undiagnosed); approximately 1 in 1,000 adults based on physician-diagnosed prevalence

Systemic Sclerosis

Systemic sclerosis (scleroderma) is a rare autoimmune disease in which the immune system triggers scarring (fibrosis) of the skin and internal organs while also damaging small blood vessels.

How common: About 276 per million U.S. adults in a large Detroit-area study; about 19 new cases per million adults each year

Waldenström Macroglobulinemia

Waldenström Macroglobulinemia is a rare blood cancer in which malignant B-lymphocytes produce excessive amounts of a protein called macroglobulin (immunoglobulin M or IgM).

How common: Approximately 1,500 new cases per year in the United States; median age of diagnosis is 68 years

Wild-Type ATTR Cardiac Amyloidosis

Wild-type ATTR cardiac amyloidosis is an age-related disease where normal transthyretin protein spontaneously misfolds and accumulates in the heart, causing progressive heart failure.

How common: Estimated to affect up to 13% of males aged 85 and older; substantially underdiagnosed

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. AstraZeneca PLC's own site is astrazeneca.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Questions about AstraZeneca PLC