Aldeyra Therapeutics Launches the Aldeyra Registry for Patients with Sjögren-Larsson Syndrome
March 1, 2017
Aldeyra Therapeutics, Inc., a biotechnology company focused on the development of products to treat diseases related to aldehydes, today announced that it has developed and launched the Aldeyra Registry for Sjögren-Larsson Syndrome (SLS), a rare inborn error of aldehyde metabolism that is characterized by severe skin and neurological disease. The Aldeyra Registry for SLS is designed to unite SLS patients worldwide, and to raise awareness of the significant physical challenges associated with the disease.
“We are delighted to collaborate with the SLS patient community on the critical task of uniting patients affected by this rare but serious disease with no FDA-approved therapy,” commented Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra. “By facilitating the degradation of the toxic fatty aldehydes that accumulate in patients with SLS, our lead compound, ADX-102, has the potential to improve the debilitating symptoms of the disease. The Aldeyra Registry is multilingual and open to SLS patients and their caregivers and doctors.”
SLS is caused by genetic mutations in Fatty Aldehyde Dehydrogenase (FALDH), an enzyme that metabolizes long-chain aldehydes. The primary day-to-day complaint of SLS patients and their caregivers is ichthyosis: severely dry, itchy skin. Patients with this debilitating disease are consistently disturbed by their dermal symptoms, which affect most of the body surface, and often excoriate their skin by scratching. There is currently no FDA-approved treatment for these symptoms. A late-stage randomized, double-blind, vehicle-controlled clinical trial of ADX-102, applied topically to the skin of SLS patients, demonstrated improvements in the severity of ichthyosis. A Phase 3 trial is expected to begin enrollment in the second half of 2017.
“The Aldeyra Registry is an important effort that can ultimately help all SLS patients. I commend Aldeyra for taking a leadership role and partnering with the SLS and rare disease patient communities,” said Dr. William Rizzo, Vice-Chair in Pediatrics at University of Nebraska Medical Center, a leading expert in SLS.
Patients, caregivers, and physicians that register will receive information about Aldeyra’s therapeutic development program in SLS, as well as opportunities to participate in clinical trials. Visit the Aldeyra Registry for SLS at: www.aldeyraregistry.com
About Aldeyra Therapeutics
Aldeyra Therapeutics, Inc. is a biotechnology company devoted to improving lives by inventing, developing and commercializing products that treat diseases thought to be related to endogenous aldehydes, a naturally occurring class of pro-inflammatory and toxic molecules. Aldeyra’s lead product candidate, ADX-102, is an aldehyde trap in development for ocular inflammation, as well as for Sjögren-Larsson Syndrome and Succinic Semi-Aldehyde Dehydrogenase Deficiency, two inborn errors of aldehyde metabolism. Aldeyra’s product candidates have not been approved for sale in the U.S. or elsewhere.
Sjögren-Larsson Syndrome (SLS)
Sjögren-Larsson Syndrome is a rare inborn error of aldehyde metabolism caused by mutations in fatty acid aldehyde dehydrogenase, leading to elevated toxic fatty aldehyde levels that are thought to contribute to severe ichthyosis (scaly, thickened, dry skin), neurological disorders, and retinal disease. There is no therapy for SLS that has been approved by the U.S. Food and Drug Administration.
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