Rare Vascular Diseases
Rare vascular diseases in the cardiovascular system consist of a collection of uncommon disorders that impact blood vessels and may disrupt the flow of blood to and from the body's organs. These conditions can affect arteries, veins, capillaries, or lymphatic vessels, resulting in a range of symptoms and potential complications. Our company aims to facilitate and expedite drug development processes by delivering essential support to researchers and scientists operating in this field and providing comprehensive preclinical development services specifically designed for rare vascular diseases.
Overview of Rare Vascular Diseases
The blood vessels carry blood to and from the body's tissues. Vascular disease affects different regions of the body and various types of blood vessels, increasing the risk of health problems due to blockage or insufficient blood flow. These conditions can lead to serious cardiovascular complications such as blood clots, heart attacks, and strokes.
Fig.1 Classification of the genes responsible for syndromic aortic diseases. (Asta, L., et al., 2023)Classification of Rare Vascular Diseases
Rare vascular diseases can be categorized into distinct groups based on the vessels affected and the underlying mechanisms. This classification may encompass arterial diseases, venous diseases, vasculitis, and other uncommon vascular abnormalities. Moreover, blood vessels play a crucial role in blood circulation, during which abnormalities can be segregated into systemic and pulmonary circulation disorders.
Rare Vascular Diseases in Systemic Circulation
Conditions in systemic circulation can be divided into various scenarios, including anatomical malformations of the arteries, autoimmune vascular diseases, spontaneous arterial dissection, premature atherosclerosis, and others.
Rare Vascular Diseases in Pulmonary Circulation
Uncommon disorders in pulmonary circulation can be organized into three main categories: congenital anomalies of the pulmonary vessels, acquired anomalies of the pulmonary vessels, and pulmonary hypertension.
Therapeutics Development for Rare Vascular Diseases
| Diseases | Drug Names | Mechanism of Action | Targets | Research Phase |
|---|---|---|---|---|
| Familial Hypercholesterolemia | Inclisiran | It reduces LDL-C levels by inhibiting the hepatic translation of proprotein convertase subtilisin/kexin type 9 (PCSK9) | PCSK9 | Approved |
| ANCA-associated Vasculitides (AAV) | Rituximab | A chimeric monoclonal antibody designed to target CD20 | CD20 | Clinical trials |
| Pulmonary Arterial Hypertension | Ataluren (PTC124) | Increasing BMPR2 protein levels | Ribosomes | Preclinical trials |
| ETRQβ-002 | Vaccine for the second extracellular loop of the endothelin-1 receptor type-A (ETAR) | ETAR | Preclinical trials |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Our Services
We understand the unique challenges associated with developing therapeutics for these conditions and are dedicated to providing customized strategies that address our client's specific needs. Our services cover a wide range of activities, including diagnostic and therapeutic development, disease modeling, pharmacokinetics, and drug safety evaluations, ensuring a seamless and effective approach to advancing projects.
Types of Rare Congenital Heart Diseases
| A-G | |
|---|---|
| ANCA-associated Vasculitides (AAV) | Familial Hypercholesterolemia |
| Antiphospholipid Syndrome | Familial Thoracic Aortic Aneurysms and Dissections |
| Chronic Thromboembolic Pulmonary Hypertension (CTEPH) | Fibromuscular Dysplasia (FMD) |
| Coarctation of the Aorta | Giant-cell Arteritis |
| Cogan Syndrome | Granulomatosis with Polyangiitis (GPA) |
| Coronary Artery Aneurysm (CAA) | Group 2 Pulmonary Hypertension |
| Coronary Artery Fistula (CAF) | |
| F-M | |
| Familial Hypercholesterolemia | Idiopathic Pulmonary Arterial Hypertension (IPAH) |
| Familial Thoracic Aortic Aneurysms and Dissections | Isolated Aortitis |
| Fibromuscular Dysplasia (FMD) | Kawasaki Disease |
| Giant-cell Arteritis | Loeys–Dietz Syndrome |
| Granulomatosis with Polyangiitis (GPA) | Marfan Syndrome |
| Group 2 Pulmonary Hypertension | Microscopic Polyangiitis (MPA) |
| Hughes-Stovin Syndrome (HSS) | |
| P-V | |
| Persistent Pulmonary Hypertension of the Newborn (PPHN) | Pulmonary Artery Voarctation |
| Polyarteritis Nodosa (PAN) | Pulmonary Capillary Hemangiomatosis (PCH) |
| Pre-eclampsia | Pulmonary Sequestration |
| Pregnancy-associated Aortic Dissection (AD) | Pulmonary Veno-occlusive disease (PVOD) |
| Primary Angiitis of the Central Nervous System (PACNS) | Right Aortic Arch |
| Primary Systemic Vasculitis | Scleroderma |
| Pulmonary Arteriovenous Fistulas (PAVFs) | Single Coronary Artery |
| Pulmonary Artery Aneurysm | Spontaneous Coronary Artery Dissection (SCAD) |
| Pulmonary Artery Dissection (PAD) | Takayasu Arteritis |
| Pulmonary Artery Sling | Vascular Ring |
Therapeutic and Animal Model Development Services
Animal Model Development Services
- Chemical-induced Model
- Diet-induced Model
- Genetically Engineered Model
- Immunogenicity Model
- Surgical Model Development
- Transplant Model Development
What differentiates our company is our profound expertise in rare cardiovascular diseases, along with advanced technologies and innovative methodologies. Our strong network of collaborators and commitment to research excellence position us as a reliable partner for developing innovative therapeutic solutions. If you're interested in our services, we encourage you to contact our team for tailored support that propels your objectives forward.
Reference
- Asta, Laura et al. "Genetic Basis, New Diagnostic Approaches, and Updated Therapeutic Strategies of the Syndromic Aortic Diseases: Marfan, Loeys-Dietz, and Vascular Ehlers-Danlos Syndrome." International journal of environmental research and public health 20.16 (2023): 6615.
For research use only, not for clinical use.