Endocrine Society

Supportive, Stable Caregiving in Childhood Protects Heart Health in Adulthood

Retrieved on: 
Tuesday, January 23, 2024
CARDIA, Hope, Pediatrics, American Heart Association, Death, Population health, NYU Langone Health, Risk, Research, Overalls, Physiology, Health, Life, Smoking, Diet, CVD, University, Lipid, Behavior, Ageing, PHD, MHS, NYU, Safety, White, Caregiver, Cardiovascular disease, Child, University of California, Circulation (journal), Popliteal artery entrapment syndrome, University of Pennsylvania, Kidney, CVH, MPH, Black, Endocrine Society, Digestive, Saint Andrew's Society, Blood pressure, Lung

New research, however, shows that receiving warmth from a caregiver during childhood protects cardiovascular health later in life, according to a study led by NYU Grossman School of Medicine and The Ohio State University Wexler Medical Center.

Key Points: 
  • New research, however, shows that receiving warmth from a caregiver during childhood protects cardiovascular health later in life, according to a study led by NYU Grossman School of Medicine and The Ohio State University Wexler Medical Center.
  • "At the same time, our findings show that adversity in early childhood does not equal destiny.
  • While adverse childhood family environments were associated with lower odds of cardiovascular health in adulthood, our findings suggest that supportive and, importantly, stable caregiving may have a stronger influence on later heart health than early adversity."
  • The hope, says Ortiz, is that this study offers insight into how supporting healthy, both supportive and stable, caregiving relationships in childhood can offer greater attainment of CVH at a population level.

AltPep Further Strengthens Advisory Board with Appointment of Steven E. Kahn, M.B., Ch.B.

Retrieved on: 
Wednesday, November 15, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Diabetes, Clinical Trials, American Diabetes Association, Amyloid, Endocrine Society, Dementia, NIH, MBA, University, Advisory board, Type 2, UW, Type 2 diabetes, Pharmaceutical industry, Medicine

AltPep Corporation, a privately held biotechnology company developing early disease-modifying treatments and detection tools for amyloid diseases, today announced the appointment of Steven E. Kahn, M.B., Ch.B.

Key Points: 
  • AltPep Corporation, a privately held biotechnology company developing early disease-modifying treatments and detection tools for amyloid diseases, today announced the appointment of Steven E. Kahn, M.B., Ch.B.
  • to its Advisory Board.
  • The Advisory Board consists of world-renowned experts in discovery, preclinical and clinical research in Alzheimer’s disease, other neurodegenerative disorders and now diabetes.
  • “We are thrilled to add Dr. Kahn’s deep clinical and research experience to our Advisory Board,” said Valerie Daggett, Ph.D., Founder and CEO of AltPep.

Visionary Professional and Healthcare Association Expert Johnnie White Named New CEO of AACE

Retrieved on: 
Thursday, December 14, 2023
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"We are extremely pleased to have Mr. White join AACE as our Chief Executive Officer," said Susan L. Samson, M.D., Ph.D., FRCPC, FACE, President-Elect of AACE and Chair of the CEO Search Committee.

Key Points: 
  • "We are extremely pleased to have Mr. White join AACE as our Chief Executive Officer," said Susan L. Samson, M.D., Ph.D., FRCPC, FACE, President-Elect of AACE and Chair of the CEO Search Committee.
  • "Mr. White brings deep leadership experience in professional associations and societies, as well as within the endocrinology specialty itself.
  • Over the coming months, Mr. White will work with the AACE Board of Directors; outgoing interim CEO Suzanne Berry, MBA, CAE; and the AACE staff to ensure an effective and seamless transition.
  • The search for AACE's new CEO was conducted in partnership with Chicago-based Tuft & Associates, Inc. and Washington, D.C.-based Association Strategies, Inc.

Lumos Pharma Reports Third Quarter 2023 Financial Results and Clinical Development Updates

Retrieved on: 
Tuesday, November 7, 2023
Research, Transgender hormone therapy, American Association of Clinical Endocrinologists, Event, Child, LUMO, Pediatric endocrinology, Therapy, City of Hope National Medical Center, Rare, IGFBP3, Mother, MD, Quarter, SDS, Endocrine Society, Clinical trial, PEM, KOL, Lumos, University, Serum, University of Chile, Political moderate, European Society of Gynaecological Oncology, Growth hormone, Investment, Consistency, AHV, Silviculture, Pharmaceutical industry, Birth control, Endocrinology

AUSTIN, Texas, Nov. 07, 2023 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, today announced that topline results met primary and secondary endpoints for its Phase 2 Dose-Finding OraGrowtH210 Trial and Phase 2 Pharmacokinetic/Pharmacodynamic (PK/PD) OraGrowtH212 Trial evaluating oral LUM-201 for subjects with moderate pediatric growth hormone deficiency (PGHD) who screened PEM-positive utilizing Lumos Pharma’s predictive enrichment marker (PEM) strategy. Lumos also announced its financial results for the quarter ended September 30, 2023.

Key Points: 
  • Lumos also announced its financial results for the quarter ended September 30, 2023.
  • The Company expects cash use of approximately $9.0 to $10.0 million in the fourth quarter of 2023.
  • Net Loss – The net loss for the quarter ended September 30, 2023 was $8.3 million compared to a net loss of $7.3 million for the same period in 2022.
  • Lumos Pharma ended the third quarter 2023 with 7,914,582 shares outstanding.

U.S. Food and Drug Administration Approves BioMarin's VOXZOGO® (vosoritide) for Children Under 5 Years with Achondroplasia

Retrieved on: 
Friday, October 20, 2023
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SAN RAFAEL, Calif., Oct. 20, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) to increase linear growth in pediatric patients with achondroplasia with open epiphyses (growth plates). This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Previously, VOXZOGO was indicated for children who were 5 years of age and older. This expanded indication now includes children of all ages with open growth plates.

Key Points: 
  • "We are pleased that VOXZOGO is now available for children of all ages with achondroplasia," said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin.
  • "We are grateful for the collaboration of the achondroplasia community, physicians, and the children and their families who have played a crucial role in advancing this clinical program.
  • The overall safety profile of VOXZOGO in children under 5 years of age was similar to that seen in older children.
  • While similar to achondroplasia, people with hypochondroplasia typically present with milder disproportionality and less severe short stature compared to achondroplasia.

Englewood Health Appoints Two New Physician Leaders to Roles in Behavioral Health and Health Equity

Retrieved on: 
Thursday, October 19, 2023
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ENGLEWOOD, N.J., Oct. 19, 2023 /PRNewswire/ -- Englewood Health has appointed two new physician leaders to oversee behavioral health and health equity.

Key Points: 
  • ENGLEWOOD, N.J., Oct. 19, 2023 /PRNewswire/ -- Englewood Health has appointed two new physician leaders to oversee behavioral health and health equity.
  • "We are delighted to welcome Dr. Spariosu as director of behavioral health," says Hillary Cohen, MD, senior vice president for medical affairs at Englewood Health.
  • Under her leadership, Englewood Health will build on its success and further meet the behavioral health needs of our communities."
  • Under her guidance, Englewood Health will implement targeted interventions designed to improve health outcomes and health equity for our communities."

BridgeBio Pharma Announces New England Journal of Medicine Publication of Positive Encaleret Proof-of-Concept Phase 2b Results in Patients with Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Retrieved on: 
Tuesday, October 10, 2023
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“Conventional therapy for ADH1 includes raising the blood calcium levels with calcium supplements and activated vitamin D, taken in multiple doses throughout day.

Key Points: 
  • “Conventional therapy for ADH1 includes raising the blood calcium levels with calcium supplements and activated vitamin D, taken in multiple doses throughout day.
  • But this burdensome regimen may also increase urine calcium levels above normal, which can damage the kidney, leading to kidney failure in worst-case scenarios.
  • People with ADH1 need better treatments, so they are not constantly walking on a tightrope.
  • As part of the Phase 2b study, participants completed one or two 5-day inpatient dose-ranging periods, followed by a 24-week outpatient period.

Lumos Pharma Announces New Data and Analysis of 15 Subjects from OraGrowtH212 Trial Presented at the 2023 ESPE Annual Meeting

Retrieved on: 
Tuesday, September 26, 2023
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“The presented results add to the growing body of evidence supporting the physiologic mechanism of action for LUM-201, as well as its potential as a treatment for moderate PGHD.

Key Points: 
  • “The presented results add to the growing body of evidence supporting the physiologic mechanism of action for LUM-201, as well as its potential as a treatment for moderate PGHD.
  • The slides presented at ESPE are available on the Posters & Publications page under the Investors & Media section of Lumos Pharma’s website.
  • At baseline, subjects (10M:5F) were (mean ± SD) aged 7.9±1.4 years, with IGF-1 SDS -0.82±0.9, and peak GH 7.2±2.2 ng/mL (clonidine stimulation), consistent with moderate PGHD.
  • Deconvolution analysis was performed on serum GH measured every 10 minutes (0800 h to 2000 h).

Rhythm Pharmaceuticals Presents New Data at ESPE 2023

Retrieved on: 
Friday, September 22, 2023
Polyphagia, LEPR, Adolescent medicine, University of Alberta, Risk, Obesity, University, Pediatrics, Metabolic syndrome, Doctor of Philosophy, Genetics, Pediatric endocrinology, Bardet–Biedl syndrome, ESPE, Endocrine Society, POMC, ROAD, BBS, Early-onset Alzheimer's disease, Patient, Division, Endocrinology, University of Ulm, Medicine, Body mass index, MC4R, European Society for Paediatric Research, Imperial College London, Proopiomelanocortin, Vaccine, Nursing, Rhythm, The Hague

BOSTON, Sept. 22, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced four oral presentations at the 61st Annual European Society for Paediatric Endocrinology (ESPE) Meeting being held September 21-23, 2023 in The Hague, Netherlands.

Key Points: 
  • BOSTON, Sept. 22, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced four oral presentations at the 61st Annual European Society for Paediatric Endocrinology (ESPE) Meeting being held September 21-23, 2023 in The Hague, Netherlands.
  • “We are excited to deliver multiple presentations at ESPE 2023 including statistics that deepen the understanding of the genetics of obesity from our Rare Obesity Advanced Diagnosis™ (ROAD) testing program as we learn more about the importance of genetics in patients with early-onset, severe obesity,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm.
  • “Frequency of Obesity-Related Gene Variants in a European Population with Early-Onset, Severe Obesity,” as presented orally by Jesús Argente M.D., Ph.D., Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid in Spain, shows that among individuals with early-onset, severe obesity, approximately 31.4% carried variants believed to be associated with obesity.
  • In addition, Rhythm is hosting a satellite symposium at ESPE 2023, titled, “Hyperphagia and early-onset, severe obesity: The role of precision medicine in the treatment of leptin melanocortin-4 receptor (MC4R) pathway diseases.”

BioMarin to Present Data Showing Long-Term Benefit of VOXZOGO® (vosoritide) on Growth in Children with Achondroplasia at 2023 European Society for Paediatric Endocrinology (ESPE) Meeting

Retrieved on: 
Thursday, September 21, 2023
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SAN RAFAEL, Calif., Sept. 21, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that new data on VOXZOGO® (vosoritide) in children with achondroplasia will be presented at the 61st Annual European Society for Paediatric Endocrinology (ESPE) Meeting in The Hague, Netherlands.

Key Points: 
  • In an open-label, long-term Phase 2 extension study, VOXZOGO demonstrated consistent and durable growth benefits in young children with achondroplasia when initiated before the age of 5.
  • Both groups of treated children demonstrated substantial restoration of height when compared to untreated children with achondroplasia.
  • VOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates.
  • VOXZOGO is also currently approved in the United States in children with achondroplasia who are 5 years of age and older with open growth plates.