Advocacy intelligence hub — real-time data for patient organizations
Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)
Arrowhead Pharmaceuticals — PHASE3
Eddingpharm (Zhuhai) Co., Ltd. — PHASE2
Beijing Suncadia Pharmaceuticals Co., Ltd — PHASE2
Arrowhead Pharmaceuticals — PHASE3
Brigham and Women's Hospital — NA
Visirna Therapeutics HK Limited — PHASE3
Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
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Praluent
Regeneron Pharmaceuticals, Inc.
Leqvio
(inclisiran)Orphan drugNovartis Pharmaceuticals Corporation
12.1 Mechanism of Action Inclisiran is a double-stranded small interfering ribonucleic acid (siRNA), conjugated on the sense strand with triantennary ...
Praluent
(alirocumab)Orphan drugRegeneron Pharmaceuticals, Inc.
PCSK9 Inhibitor [EPC]
12.1 Mechanism of Action Alirocumab is a human monoclonal antibody that binds to proprotein convertase subtilisin kexin type 9 (PCSK9). PCSK9 binds to...
Evkeeza
(evinacumab-dgnb)Orphan drugRegeneron Pharmaceuticals, Inc.
Angiopoietin-like 3 Inhibitor [EPC]
12.1 Mechanism of Action Evinacumab-dgnb is a recombinant human monoclonal antibody that binds to and inhibits ANGPTL3. ANGPTL3 is a member of the ang...
Crestor
(rosuvastatin)Orphan drugiPR Pharmaceuticals, Inc.
12.1 Mechanism of Action CRESTOR is an inhibitor of HMG‑CoA reductase, the rate-limiting enzyme that converts 3‑hydroxy‑3‑methylglutaryl coenzyme A to...
Repatha
(evolocumab)Orphan drugAmgen Inc.
PCSK9 Inhibitor [EPC]
12.1 Mechanism of Action Evolocumab is a human monoclonal IgG2 directed against human proprotein convertase subtilisin kexin type 9 (PCSK9). PCSK9 bin...
KYNAMRO
(mipomersen)Orphan drugKastle Therapeutics, LLC
Juxtapid
(lomitapide)Orphan drugChiesi Farmaceutici S.p.A.
12.1 Mechanism of Action JUXTAPID directly binds and inhibits microsomal triglyceride transfer protein (MTP), which resides in the lumen of the endopl...
A case of presumed homozygous familial hypercholesterolemia.
We present a case of a young adult diagnosed with homozygous familial hypercholesterolemia despite a normal lipid panel. A deeper dive into the details of the genotype resulted in recharacte...
LDL-C target achievement after adding evinacumab in 2 patients with autosomal recessive hypercholesterolemia.
Autosomal recessive hypercholesterolemia (ARH) is a rare form of genetic hypercholesterolemia consequent to pathogenic variants in the low-density lipoprotein receptor adaptor protein 1 (LDLRAP1) ...
50 years of apheresis for homozygous familial hypercholesterolemia.
50 years of apheresis for homozygous familial hypercholesterolemia.
Novel Approaches to Lipid Management: Beyond Statins and PCSK9 Inhibitors.
The statins remain the foundation of lipid management because they lower low-density lipoprotein cholesterol (LDL-C) and prevent cardiovascular events, and guidelines recommend stepwise intensificatio...
Evinacumab with or without lipoprotein apheresis in homozygous familial hypercholesterolaemia.
Homozygous familial hypercholesterolaemia (HoFH) is characterized by markedly elevated low-density lipoprotein cholesterol (LDL-C). Most individuals with HoFH do not reach LDL-C targets with standard ...
The impact of antisense oligonucleotide (ASO) therapeutics on the future of rare disease drug discovery
Published in Expert Opin Drug Discov. Ruchi R et al.
Patrick Couture, MD, FRCP, PhD
Laval University
📍 NASHVILLE, TN
Dan J Rader, MD, MD
University of Pennsylvania
📍 PHILADELPHIA, PA
Evan A Stein, MD PhD, MD
LIB Therapeutics
📍 CHICAGO, IL
Marina Cuchel, MD, PhD
University of Pennsylvania
Mark Sumeray, MD
Aegerion Pharmaceuticals, Inc.
Mariko Harada-Shiba, M.D., Ph.D.
National Cerebral and Cardiovascular Center Research Institute
View all Homozygous familial hypercholesterolemia specialists →