Turner syndrome

Ascendis Pharma Reports First Quarter 2024 Financial Results

Retrieved on: 
Thursday, May 2, 2024
FDA, Endocrine gland, Hypoparathyroidism, Element, GHD, Achondroplasia, Commercial, New Drug Application, Biologics license application, Development, Prescription, ASCO, Turner syndrome, PDUFA, Patient, Society, Melanoma, Vision, Inborn errors of metabolism, Pharmaceutical industry

If approved, U.S. launch planned in Q3

Key Points: 
  • If approved, U.S. launch planned in Q3
    — SKYTROFA® Q1 revenue more than doubled year-over-year to €65 million; Q1 operating expenses fell by 20% year-over-year to €137 million
    COPENHAGEN, Denmark, May 02, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced financial results for the first quarter ended March 31, 2024, and provided a business update.
  • Plan to submit a supplemental Biologics License Application to FDA for adult growth hormone deficiency (GHD), in the third quarter of 2024.
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • In the U.S., Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if approved, U.S. commercial launch planned in the third quarter of 2024.

Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Fragile X syndrome, Civil service commission, Diagnosis, X chromosome, Brain, FMRP, Woman, Learning disability, Disease, Nervous system, Patient, Committee, Knowledge, History, Regulation, Traffic barrier, Anxiety, EMA, IRIS, Mouth, European, COMP, European Commission, Consultant, Safety, Marketing, DSM-IV codes, Turner syndrome, Medicine, Pharmaceutical industry

Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

Key Points: 


Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

North America Molecular Diagnostics Market Size, Share & Trends Analysis Report 2024-2030: Growing Prevalence of Target Diseases & Increasing External Funding for R&D Fueling Expansion - ResearchAndMarkets.com

Retrieved on: 
Tuesday, March 26, 2024
Health, Biotechnology, Candidiasis, Diagnosis, PCR, MVP, Conditional sentence, Bacterial vaginosis, Prevalence, Infection, Promega, Country, Thermo Fisher Scientific, SME, Tuberculosis, Application, ISH, RT, HIV, Alzheimer's disease, Incidence, Turner syndrome, Cepheid variable, Research, Trichomoniasis, Real-time, Cancer, Death, Parkinson's disease, CDC, QIAGEN, Therapy, USD, Horvitz–Thompson estimator, Helix, DSM-IV codes, Vaccine

Advancements in molecular diagnostic technology enabled the early detection of numerous diseases and reduced the possibility of severe economic & social burdens.

Key Points: 
  • Advancements in molecular diagnostic technology enabled the early detection of numerous diseases and reduced the possibility of severe economic & social burdens.
  • Molecular diagnostics enable early diagnosis of cancer, genetic disorders, and infectious diseases by using PCR, sequencing, & genetic technologies.
  • In addition, the increasing incidence of diseases, such as influenza A & B, is projected to drive market growth.
  • An increase in external funding to conduct clinical studies in molecular diagnostics is anticipated to fuel the market.

Ascendis Pharma Reports Fourth Quarter and Full Year 2023 Results

Retrieved on: 
Wednesday, February 7, 2024
Investigational New Drug, Oncology, Endocrine gland, Research, GHD, Patient, New Drug Application, CNP, FDA, Achondroplasia, PDUFA, Turner syndrome, Webcast, Pharmaceutical industry

Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.

Key Points: 
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • During the fourth quarter of 2024, plan to submit an Investigational New Drug application or similar in adults with achondroplasia.
  • Total revenue for the fourth quarter of 2023 was €137.7 million compared to €22.9 million during the same period for 2022.
  • Ascendis Pharma will also host a conference call and webcast today at 4:30 p.m. Eastern Time (ET) to discuss 2023 financial results.

Ascendis Pharma Introduces Vision 2030

Retrieved on: 
Sunday, January 7, 2024
Growth hormone, Vision 2030, Patient, Endocrine gland, Hypoparathyroidism, Conference, Achondroplasia, Turner syndrome, FDA, GHD, PDUFA, Globalization, Investigational New Drug, New Drug Application, Transgender hormone therapy, J. P. Morgan, Ophthalmology, CNP, HNSCC, Growth, CTA, Pharmaceutical industry

COPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision 2030, its strategic roadmap through 2030. Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 8, at the 42nd Annual J.P. Morgan Healthcare Conference.

Key Points: 
  • COPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision 2030, its strategic roadmap through 2030.
  • Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 8, at the 42nd Annual J.P. Morgan Healthcare Conference.
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at https://investors.ascendispharma.com .

HSS Study Reveals Concerning Link Between Growth Hormone Therapy and Growth Plate Fractures in Children

Retrieved on: 
Thursday, January 4, 2024
Growth hormone, Patient, Quadriceps, Injury, Hospital, Food, FACS, Fracture, Turner syndrome, Risk, Adolescence, Hospital Records, Research, Prader–Willi syndrome, Hospital for Special Surgery, Tibia, HSS, Motion, Parent, Kidney disease, MPH, Journal, Child, Dentistry

NEW YORK, Jan. 4, 2024 /PRNewswire/ -- Human growth hormone has long been used to treat certain medical conditions in children. New research from surgeon-scientists at Hospital for Special Surgery (HSS) shows that there may be a previously unknown complication.

Key Points: 
  • In children, human growth hormone therapy has been used for decades to treat growth hormone deficiency due to underlying medical conditions such as Turner syndrome, very small weight at birth, Prader-Willi syndrome and chronic renal insufficiency.
  • Given the volume of patients we treat, we were able to investigate the relationship between tibia growth plate fractures and growth hormone therapy in pediatric patients."
  • Therefore, the odds of taking growth hormone therapy were 15 times higher among patients with proximal tibia physeal avulsion fractures than the comparison group.
  • "Further research is required to tease out whether limiting specific high-impact sports will mitigate the risk of tibia growth plate fractures in active children and adolescents."

Insights Revealed in 2023 Turner Syndrome Pipeline Report: 4+ Companies and 4+ Promising Drugs in Focus - ResearchAndMarkets.com

Retrieved on: 
Tuesday, July 25, 2023
Genetics, Pharmaceutical, Health, Drug development, Therapy, Turner syndrome, Pituitary gland, Research, GH, Endocrine gland, X chromosome, Growth hormone, Chromosome, Patient, Southern Transcon, Ibutamoren, Pharmaceutical industry

This report presents comprehensive insights into the Turner Syndrome pipeline landscape, featuring 4+ companies and 4+ pipeline drugs.

Key Points: 
  • This report presents comprehensive insights into the Turner Syndrome pipeline landscape, featuring 4+ companies and 4+ pipeline drugs.
  • Turner Syndrome, also known as congenital ovarian hypoplasia syndrome, is the most common sex chromosomal abnormality found in females.
  • In most cases, Turner Syndrome is not inherited and results from a random event during the formation of reproductive cells.
  • Apart from Lumos Pharma and Ascendis Pharma, the report covers other companies actively engaged in developing therapies for Turner Syndrome.

Ascendis Pharma Showcases Its Latest Endocrinology Programs, Data, and Research at ENDO 2023

Retrieved on: 
Tuesday, June 13, 2023
ENDO, Multicenter trial, Turner syndrome, Hypoparathyroidism, Pathway, Efficacy, Saint Andrew's Society, Research, Human Accomplishment, Southern Transcon, Randomness, Endocrine Society, Poster, Pharmaceutical industry, Achondroplasia, Tolerability, Safety, Endocrinology

COPENHAGEN, Denmark, June 13, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it will showcase its latest Endocrinology Rare Disease programs, data, and research in growth hormone deficiency, hypoparathyroidism, achondroplasia, and Turner Syndrome at ENDO 2023, the annual meeting of the Endocrine Society being held June 15-18 in Chicago.

Key Points: 
  • - Endocrinology clinical development progress and research in growth hormone deficiency, hypoparathyroidism, achondroplasia, and Turner Syndrome will be highlighted
    COPENHAGEN, Denmark, June 13, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it will showcase its latest Endocrinology Rare Disease programs, data, and research in growth hormone deficiency, hypoparathyroidism, achondroplasia, and Turner Syndrome at ENDO 2023, the annual meeting of the Endocrine Society being held June 15-18 in Chicago.
  • An oral presentation on Saturday, June 17, will feature Phase 3 Week 52 data for TransCon PTH in adults with hypoparathyroidism, and five additional poster presentations listed in the table below will showcase other Ascendis programs and data.
  • In addition, Ascendis will host booth #1628, booth #1740, and two product theaters during ENDO 2023.
  • Long-Term Efficacy and Safety of TransCon PTH in Adults with Hypoparathyroidism: 52-Week Results from the Open-Label Extension of the PaTHway Phase 3 Trial
    Clinical and Economic Burden of Postsurgical Chronic Hypoparathyroidism: A U.S. Medicare Retrospective Analysis
    Significantly Improved Annual Height Velocity with Once-Weekly TransCon CNP in Children with Achondroplasia: The ACcomplisH Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose-Escalation Trial
    Design of the New InsiGHTS Trial: A Multicenter, Phase 2, Randomized, Open-Label, Active Controlled Study to Investigate the Safety, Tolerability, and Efficacy of Lonapegsomatropin in Prepubertal Individuals with Turner Syndrome

Devyser launches first IVDR-certified product

Retrieved on: 
Wednesday, March 1, 2023
Certification, IVDR, CCO, Laboratory, Risk, Turner syndrome, Diagnosis, Chromosome, Medical device

STOCKHOLM, March 1, 2023 /PRNewswire/ -- Devyser, the pioneering leader in diagnostic solutions, launches today its first IVDR-certified product.

Key Points: 
  • STOCKHOLM, March 1, 2023 /PRNewswire/ -- Devyser, the pioneering leader in diagnostic solutions, launches today its first IVDR-certified product.
  • The original product was first launched in 2006 and is today the golden standard in prenatal testing in many European countries.
  • To our customers it´s a reassurance that we will continue to provide safe and reliable products", says Theis Kipling, CCO, Devyser.
  • The certification was issued by the notified body TÜV SÜD in August 2022 and covers Devyser Compact and Devyser´s quality management system.

JUNO DIAGNOSTICS™ EXPANDS ITS PRODUCT PORTFOLIO WITH THE LAUNCH OF JUNO HAZEL™ PLUS AND ANNOUNCES EARLY ACCESS PROGRAM (EAP) FOR INNOVATIVE NIPS TESTS

Retrieved on: 
Monday, January 9, 2023
Klinefelter syndrome, Turner syndrome, Blood, XYY syndrome, Commercial Operating System (COS), Prenatal care, Genetic testing, SAN, Phlebotomy, EAP, XXY, Pregnancy, Patient, Hazel, Edwards, Special access program, Physician, NIPT, XYY, NIPS, Trisomy, Down syndrome, Edwards syndrome, Genetic counseling, Juno, Pharmaceutical industry

Similar to the Juno Hazel™ screening test, Juno Hazel™ Plus leverages JunoDx's proprietary Sample Collection Kit to improve early access to high-quality genetic testing without the high costs, long lead times, and phlebotomy requirements of venous-based NIPS.

Key Points: 
  • Similar to the Juno Hazel™ screening test, Juno Hazel™ Plus leverages JunoDx's proprietary Sample Collection Kit to improve early access to high-quality genetic testing without the high costs, long lead times, and phlebotomy requirements of venous-based NIPS.
  • Juno Hazel™ Plus offers a comprehensive prenatal screening solution by screening for common chromosomal trisomies, sex chromosome aneuploidies, and fetal sex.
  • "The expansion of our product portfolio with the launch of our second NIPS test , Juno Hazel™ Plus, represents a significant milestone for JunoDx™.
  • To ensure every pregnancy has the opportunity to benefit from our product portfolio, the time is now to create and launch a formalized Early Access Program.