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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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Latest News

NewLink Genetics and Lumos Pharma Enter into Merger Agreement to Form Biopharmaceutical Company Focused on Developing Therapies to Treat Rare Diseases

— Combined company to assume the name, Lumos Pharma, Inc. and is expected to be led by Richard J. Hawkins, current CEO of Lumos Pharma — — Lumos asset, LUM-201, represents first oral therapeutic candidate for pediatric growth hormone deficiency (PGHD) with planned Phase 2b expected to begin in mid-2020 — — Lumos Pharma stockholders,…

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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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