OUR FOCAL POINTS:
Pediatric Growth Hormone Deficiency (PGHD)
PGHD results in growth retardation, short stature and maturation delays whose origin might be congenital, acquired or idiopathic.
Creatine Transporter Deficiency
CTD is a genetic disease, an inborn error of metabolism, that appears in early childhood and often results in severe intellectual disability.
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…READ MORE
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…READ MORE
10 CTD Study Sites Now Open
The Vigilan Study – Observational Study of Creatine Transporter Deficiency (CTD) of Males, now has ten study sites open for patient enrollment. Males with CTD will be screened at university clinics. Once enrolled in the study, participants will undergo a series of age-appropriate tests of physical and intellectual abilities. Follow-up testing will occur every 6…READ MORE