Vascular Ehlers-Danlos Syndrome Patient Data Released Following Study

According to a story from globenewswire.com, the pharmaceutical company Acer Therapeutics recently announced the release of data from a long-term observational study of patients with vascular Ehlers-Danlos syndrome (vEDS). All of the patients in this study tested positive for a COL3A1 mutation, which is one of several mutations linked to Ehlers-Danlos syndrome and causes fundamental changes to fibrous proteins. Acer is committed to the development of treatments for rare, serious, life-threatening conditions and diseases.

About Ehlers-Danlos Syndrome

Ehlers-Danlos syndrome is a group of disorders that affect connective tissue. There are several different types which have varied symptoms and are linked to different genetic mutations. These mutations may be inherited or may appear early in a patient’s development. Symptoms can include loose joints, stretchy skin, scarring, pain, aoritic dissection, scoliosis, and osteoarthritis. Vascular Ehlers-Danlos syndrome is linked to mutations of the COL3A1 gene and is characterized by fragile, transparent skin, thin hair, short stature, fragile organs prone to rupture, distinct facial features, club foot, and joint hypermobility. With no cure currently available, the treatment of these syndromes is supportive and based on symptoms as they appear. Surgery may be utilized to aid joint problems, but results are varied. There is a great disparity in the severity of symptoms; some patients are able to lead fairly normal lives, whereas others may be severely affected. Vascular Ehlers-Danlos syndrome has a median life expectancy of just 48 years. To learn more about Ehlers-Danlos syndrome, click here.

About The Study

This long term study includes data from 144 patients with vascular Ehlers-Danlos syndrome. The data included was from patients who received treatment from 2000 to 2017. The research found that half of patients were not receiving regular treatment at their first check up. By the conclusion of the data collection period, 90 percent of patients were being treated with the medication celiprolol. 62.5 percent of patients were using the maximum tolerable dose during their follow up.

The study findings highlight the effectiveness of celiprolol, a drug developed by Acer Therapeutics, in improving patient survival. After an 11.1 year follow up, survival was 80.7 percent for patients that were regularly using the drug, but only 48.5 percent in those patients that were not. 


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