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Pediatric Growth Hormone Deficiency (PGHD)

PGHD results in growth retardation, short stature and maturation delays whose origin might be congenital, acquired or idiopathic.


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Transferring Sponsorship of a Clinical Study: A lesson on how rare disease drug developers can work together for the good of an underserved rare disease community

    On June 20th, 2019, wonderful news was announced on the Association for Creatine Deficiencies’ (ACD) website and ACD Facebook page that Lumos Pharma has transferred the sponsorship role of the Vigilan Study (Observational Study of Creatine Transporter Deficiency-CTD) to Ultragenyx Pharmaceutical Company. https://creatineinfo.org/wp-content/uploads/2019/06/Vigilan-Update-June-20-2019-Transfer-ACD-Letter.pdf “The ongoing natural history study, Vigilan, is crucial for the…

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IT’S RARE DISEASE DAY AND WE ARE ADVANCING CTD KNOWLEDGE!

By Carol A. Dutch – Senior Director, Patient Engagement, Lumos Pharma More than two years ago, a multidisciplinary team of investigators based primarily at Children’s Hospital of Philadelphia (CHOP) set out to advance the scientific and medical community’s understanding of a rare pediatric neurodevelopmental disorder called Creatine Transporter Deficiency (CTD/SLC6A8), first identified in 2001. Their work culminates…

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Lumos Pharma Acquires Candidate for Oral Treatment of Growth Hormone Deficiency

Lumos Pharma, Inc., a clinical stage biopharmaceutical company focused on development and commercialization of therapeutics for rare and neglected diseases, today announced that it has acquired the license for LUM-201, an investigational orally administered small molecule that promotes secretion of growth hormone from the pituitary gland, from Ammonett Pharma LLC.  Lumos plans to initiate a…

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