The INADcure Foundation does not provide financial support to the following study.
Retrotope, Inc. Assessing medical records of children with INAD in anticipation of FDA approval of clinical trail.
Retrotope, Inc. is currently assessing medical records of children with INAD in anticipation of FDA approval for an upcoming clinical trial for its chemically-modified polyunsaturated fatty acid drug (RT001) for the treatment of PLA2G6 associated neurodegeneration (PLAN).
About Retrotope – Retrotope, a privately-held, clinical-stage pharmaceutical company, is creating a new category of drugs to treat degenerative diseases. Composed of proprietary compounds that are chemically stabilized forms of essential nutrients, these compounds are being studied as disease modifying therapies for many intractable diseases such as Parkinson’s, Alzheimer’s, neuromuscular diseases, and retinopathies. RT001, Retrotope’s first lead candidate, is for the treatment of rare orphan neuromuscular diseases, and is being tested in clinical trials for Friedreich’s ataxia and INAD. For more information about Retrotope, please visit www.retrotope.com.
About RT001 – RT001 is a patented, orally available modified fatty-acid therapeutic that stabilizes (“fireproofs”) mitochondrial and cellular membranes against attack and restores cellular health. Retrotope has discovered that lipid peroxidation, the free-radical degradation of lipids in mitochondrial and cellular membranes, may be causative of a wide range of degenerative diseases. Free radicals attack and degrade polyunsaturated fats (PUFAs) that are essential membrane components. Retrotope has shown that the degradation products of these fats create toxic cascades in many illnesses of degeneration.
March 2017 – Retrotope enrolled its first patient compassionate use trial for its chemically-modified polyunsaturated fatty acid drug (RT001) for the treatment of PLA2G6 associated neurodegeneration (PLAN).
November 2, 2017 – Retrotope announced that the U.S. Food and Drug Administration’s (FDA’s) Office of Orphan Products Development (OOPD) granted orphan drug designation for its chemically-modified polyunsaturated fatty acid drug (RT001) for the treatment of PLA2G6 associated neurodegeneration (PLAN).
See full press release here: https://static1.squarespace.com/static/549af14ae4b004237f7bb71a/t/5a0ea01d085229ac6656c5d5/1510907934034/Retrotope+INAD+Orphan+Drug+Press+11-2-2017+final+wire.pdf
November 16, 2017 – Retrotope enrolled a second subject in a compassionate use trial of the ultra-rare, neurological disease, Infantile Neuroaxonal Dystrophy (INAD).
See full press release here: https://static1.squarespace.com/static/549af14ae4b004237f7bb71a/t/5a0ea1b3e4966bd1984dc50f/1510908340401/Retrotope+INAD+Patient+2+Final+Press+11-16-2017.pdf
For INAD Patients and Families: https://www.retrotope.com/inad-clinical-trial
For additional information please direct all questions to:
Senior Director of Clinical Operations
(408) 834- 5729