Health
Drug shows potential to slow progression of rare, painful genetic disease
NIH-supported clinical trial could lead to first effective treatment for ACDC disease
what: Drugs used to treat certain bone diseases slow the progression of a rare, painful genetic disease known as arterial calcification due to CD73 deficiency (ACDC), which causes excess calcium to build up in the arteries. It is expected that. These results come from a first-in-human clinical trial supported by the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.Research published in journals vascular medicine, It could lead to the first effective treatment for a rare disease.
ACDC has no known cure, but it often targets arteries in the legs and can cause pain and difficulty walking. It can also affect the joints of the hand, causing pain and deformity. In severe cases, this condition can lead to limb loss. Symptoms of this disease often begin in the late teens or 20s. It is a very rare disease, thought to affect only about 20 people worldwide, with a prevalence estimated at less than 1 in 1 million people. Previous studies have identified the genes and underlying biochemical mechanisms of ACDC disease. A recent study by the NHLBI research team identified an existing drug called . etidronic acidas a potential treatment for ACDC based on animal and human cell disease models.
In this study, researchers evaluated the safety and efficacy of etidronate in treating arterial calcification and impaired blood flow in the legs in seven people (four women and three men) with ACDC disease. Although small in number, together they account for about one-third of known cases worldwide. Treatment consisted of taking oral medication daily for 14 days every 3 months for 3 years. Researchers used CT scans to measure calcium deposits and examined blood flow using a non-invasive tool, the ankle-brachial index, both at the beginning of the study and at annual follow-up after treatment. did.
Researchers found that etidronate treatment appeared to be safe, with no adverse side effects reported. This drug appears to slow the development of new calcium deposits in blood vessels in the legs and also slow the progression of blood flow obstruction. However, the drug did not reverse the calcium deposits that were already present in the affected blood vessels or joints, nor did it show any obvious improvement in blood flow. Questionnaires administered to patients suggested improvements in symptoms such as pain and movement disorders.
The researchers suggest that lessons learned from the current study may enable the development of new treatments for ACDC and large-scale clinical trials for the disease. This study may also shed light on other diseases associated with excess calcium buildup in the arteries. peripheral artery disease (pad) and atherosclerosis.
reference: The paper “Pilot study to assess the safety and efficacy of etidronate treatment for arterial calcification due to CD73 deficiency (ACDC)” vascular medicine.
Who: Elisa Ferrante, PhD, clinical program manager and staff scientist in NHLBI's Division of Translational Vascular Medicine, and Alessandra Brofferio, MD, clinical cardiologist in NHLBI's Institute for Cardiovascular Regenerative Medicine.
|
Sources 2/ https://www.nhlbi.nih.gov/news/2024/drug-shows-promise-slowing-progression-rare-painful-genetic-disease The mention sources can contact us to remove/changing this article |
What Are The Main Benefits Of Comparing Car Insurance Quotes Online
LOS ANGELES, CA / ACCESSWIRE / June 24, 2020, / Compare-autoinsurance.Org has launched a new blog post that presents the main benefits of comparing multiple car insurance quotes. For more info and free online quotes, please visit https://compare-autoinsurance.Org/the-advantages-of-comparing-prices-with-car-insurance-quotes-online/ The modern society has numerous technological advantages. One important advantage is the speed at which information is sent and received. With the help of the internet, the shopping habits of many persons have drastically changed. The car insurance industry hasn't remained untouched by these changes. On the internet, drivers can compare insurance prices and find out which sellers have the best offers. View photos The advantages of comparing online car insurance quotes are the following: Online quotes can be obtained from anywhere and at any time. Unlike physical insurance agencies, websites don't have a specific schedule and they are available at any time. Drivers that have busy working schedules, can compare quotes from anywhere and at any time, even at midnight. Multiple choices. Almost all insurance providers, no matter if they are well-known brands or just local insurers, have an online presence. Online quotes will allow policyholders the chance to discover multiple insurance companies and check their prices. Drivers are no longer required to get quotes from just a few known insurance companies. Also, local and regional insurers can provide lower insurance rates for the same services. Accurate insurance estimates. Online quotes can only be accurate if the customers provide accurate and real info about their car models and driving history. Lying about past driving incidents can make the price estimates to be lower, but when dealing with an insurance company lying to them is useless. Usually, insurance companies will do research about a potential customer before granting him coverage. Online quotes can be sorted easily. Although drivers are recommended to not choose a policy just based on its price, drivers can easily sort quotes by insurance price. Using brokerage websites will allow drivers to get quotes from multiple insurers, thus making the comparison faster and easier. For additional info, money-saving tips, and free car insurance quotes, visit https://compare-autoinsurance.Org/ Compare-autoinsurance.Org is an online provider of life, home, health, and auto insurance quotes. This website is unique because it does not simply stick to one kind of insurance provider, but brings the clients the best deals from many different online insurance carriers. In this way, clients have access to offers from multiple carriers all in one place: this website. On this site, customers have access to quotes for insurance plans from various agencies, such as local or nationwide agencies, brand names insurance companies, etc. "Online quotes can easily help drivers obtain better car insurance deals. All they have to do is to complete an online form with accurate and real info, then compare prices", said Russell Rabichev, Marketing Director of Internet Marketing Company. CONTACT: Company Name: Internet Marketing CompanyPerson for contact Name: Gurgu CPhone Number: (818) 359-3898Email: [email protected]: https://compare-autoinsurance.Org/ SOURCE: Compare-autoinsurance.Org View source version on accesswire.Com:https://www.Accesswire.Com/595055/What-Are-The-Main-Benefits-Of-Comparing-Car-Insurance-Quotes-Online View photos
to request, modification Contact us at Here or [email protected]
 |
 |
 |
