Polydactyly

Rhythm Pharmaceuticals Announces Positive Reimbursement Decision in Italy for IMCIVREE® (setmelanotide) for the Treatment of Obesity and Control of Hunger in Bardet-Biedl Syndrome

Retrieved on: 
Wednesday, February 7, 2024
Visual impairment, European, Deficit spending, International, Polydactyly, Obesity, POMC, LEPR, Patient, Prevalence, BBS, Polyphagia, Gazzetta Ufficiale, Rhythm, Hunger, Medicine, MC4R, PCSK1, Bardet–Biedl syndrome, Hypogonadism, Pharmaceutical industry

“We are pleased that AIFA has recognized the need for a precision medicine for obesity and control of hunger in BBS,” said Yann Mazabraud, Executive Vice President and Head of International at Rhythm.

Key Points: 
  • “We are pleased that AIFA has recognized the need for a precision medicine for obesity and control of hunger in BBS,” said Yann Mazabraud, Executive Vice President and Head of International at Rhythm.
  • BBS is also associated with cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment.
  • In Italy, the Company estimates the prevalence of BBS to be between 1/125,000 and 1/160,000, with approximately 200 identified.
  • AIFA previously approved reimbursement for IMCIVREE for the treatment of obesity and control of hunger associated with biallelic proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or biallelic leptin receptor (LEPR) deficiency.

Rhythm Pharmaceuticals Announces Completion of Screening for Enrollment in Setmelanotide Phase 3 Hypothalamic Obesity Trial and Additional Updates

Retrieved on: 
Thursday, January 4, 2024
Bardet–Biedl syndrome, Hyperpigmentation, Medicine, Deficit spending, IND, Polyphagia, FDA, Polydactyly, Prevalence, POMC, International, Visual impairment, Syndrome, Investigational New Drug, Ministry, Obesity, PCSK1, Patient, Hunger, Hypogonadism, BBS, LEPR, Pharmaceutical industry

BOSTON, Jan. 04, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today provided a business update and announced it has completed screening for enrollment in the pivotal, Phase 3 clinical trial evaluating setmelanotide in patients with acquired hypothalamic obesity.

Key Points: 
  • Rhythm closed screening and anticipates overenrolling the ongoing phase 3 hypothalamic obesity trial with more than 140 patients consented and going through active screening and baseline assessments.
  • In this trial, patients with acquired hypothalamic obesity aged 4 years or older will be randomized 2:1 to setmelanotide therapy or placebo for a total of 60 weeks, including up to eight weeks for dose titration.
  • Rhythm estimates the prevalence of BBS to be approximately 700 people in Spain with approximately 100 patients identified.
  • The Company anticipates beginning Phase 1 in-human trials in the first half of 2024, including a multiple-ascending dose study in patients with hypothalamic obesity.

Rhythm Pharmaceuticals Receives Positive CADTH Reimbursement Recommendation for IMCIVREE® (setmelanotide)

Retrieved on: 
Thursday, November 2, 2023
DSM-IV codes, Faculty, Hypogonadism, BBS, BMI, Obesity, Life, CADTH, Prevalence, Health Canada, News, Deficit spending, Nausea, Bardet–Biedl syndrome, Priority review, Polyphagia, Visual impairment, Disease, Medicine, Body mass index, University, MC4R, University of Melbourne Faculty of Medicine, Dentistry and Health Sciences, Polydactyly, University of Alberta, Patient, Pharmaceutical industry, Health

BOSTON, Nov. 02, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global 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 that the Canadian Agency for Drugs and Technologies in Health (CADTH) has recommended that IMCIVREE® (setmelanotide) be reimbursed by CADTH-participating public drug plans, with conditions, for weight management in adult and pediatric patients six years of age and older with obesity due to Bardet-Biedl syndrome (BBS).

Key Points: 
  • People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life.
  • BBS may also be associated with cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment.
  • “This recommendation represents a significant milestone for Canadian patients living with obesity due to BBS.
  • We appreciate CADTH’s recommendation and their recognition of the rare genetic nature of BBS, the unmet needs of these patients and that IMCIVREE is the only available therapy that targets the underlying disease mechanism of BBS,” said Carol Stiff, General Manager of Rhythm Pharmaceuticals Canada Inc.

Rhythm Pharmaceuticals Announces New ICD-10 Diagnosis Code Established for Bardet-Biedl Syndrome

Retrieved on: 
Wednesday, September 6, 2023
Polyphagia, Obesity, International Classification of Diseases, Prevalence, Bardet–Biedl syndrome, Deficit spending, Visual impairment, Knowledge Organization (journal), Brain, CDC, BBS, HIV disease progression rates, Patient, Physician, Syndrome, Korea Disease Control and Prevention Agency, Hypothalamus, MC4R, Diagnosis, Hunger, News, Hypogonadism, Polydactyly, Disease, Pharmaceutical industry, Nursing

The new code, Q87.83 Bardet-Biedl syndrome (BBS), takes effect Oct. 1, 2023.

Key Points: 
  • The new code, Q87.83 Bardet-Biedl syndrome (BBS), takes effect Oct. 1, 2023.
  • “Finding and connecting patients, families and physicians across the country has been extremely challenging because of the lack of a dedicated ICD-10 code as patients with BBS are lumped in with many other genetic syndromes,” said Tim Ogden, President of the BBS Foundation.
  • “We believe this new disease specific ICD-10 code marks an important advance for the care of patients with BBS,” said Jennifer Chien, Rhythm Executive Vice President and Head of North America.
  • People living with BBS also may experience cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment.

Rhythm Pharmaceuticals Announces Commercial Launch of IMCIVREE® (setmelanotide) in Germany for the Treatment of Obesity and Control of Hunger in Bardet-Biedl Syndrome

Retrieved on: 
Monday, April 24, 2023
Polyphagia, LEPR, Obesity, International, Prevalence, Life, Food and Drug Administration, Bardet–Biedl syndrome, Rhythm, Deficit spending, Polydactyly, Visual impairment, DSM-IV codes, POMC, BBS, Very early onset inflammatory bowel disease, Patient, PCSK1, National Association of Statutory Health Insurance Physicians, Medicine, European, Endocrinology, MC4R, FDA, EC, Hunger, Hypogonadism, Food, European Commission, Pharmaceutical industry, EU

The German Federal Joint Committee (G-BA) ruled that IMCIVREE is eligible for reimbursement by Statutory Health Insurances for BBS.

Key Points: 
  • The German Federal Joint Committee (G-BA) ruled that IMCIVREE is eligible for reimbursement by Statutory Health Insurances for BBS.
  • People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life.
  • In 2022, Rhythm was granted marketing authorization for IMCIVREE by the EC for BBS and early-access authorization for setmelanotide in France.
  • Rhythm expects to launch IMCIVREE for the treatment of patients with BBS in the Netherlands later this year and in Italy, Spain, and the UK in 2024.

Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trial of IMCIVREE® (setmelanotide) in Bardet-Biedl Syndrome in The Lancet Diabetes and Endocrinology

Retrieved on: 
Tuesday, November 8, 2022
People, GLOBE, Polyphagia, BBS, Government budget balance, Adult, MC4R, Visual impairment, SD, Weight, University of Alberta, Nasdaq, Hypogonadism, Bardet–Biedl syndrome, Department, BMI, Faculty, Very early onset inflammatory bowel disease, Obesity, Hunger, Patient, Safety, Weight loss, University of Melbourne Faculty of Medicine, Dentistry and Health Sciences, University, Life, The Lancet, Polydactyly, Medicine, Pharmaceutical industry, Endocrinology, EU

BOSTON, Nov. 08, 2022 (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 that previously disclosed results from a Phase 3 clinical trial that evaluated IMCIVREE® (setmelanotide), an MC4R agonist, in patients with Bardet-Biedl syndrome (BBS) have been published in the peer-reviewed journal The Lancet Diabetes and Endocrinology. The trial met its primary endpoint and all key secondary endpoints, with statistically significant reductions in weight and hunger at 52 weeks on therapy.

Key Points: 
  • People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life.
  • Rhythms multicenter Phase 3 trial (NCT03746522) enrolled patients 6 years old with obesity and BBS or Alstrm syndrome (N=38).
  • The primary endpoint was the proportion of patients 12 years old in the full analysis set achieving 10% weight reduction after 52 weeks.
  • As previously disclosed, treatment with setmelanotide resulted in significant weight and hunger reductions after one year of treatment among patients with BBS.

Global Drug Taste Masking and Taste Assessment Services and Technologies Market Report 2022-2035 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, July 26, 2022
Health, Pharmaceutical, Tongue, Capsule, Population, Type, Industry, Food, EMA, Partnership, Compliance, US, USD, International Flavors & Fragrances, US FDA, Operation, Taste, US Foods, Patient, Polydactyly, IFF, European Medicines Agency, Growth, School of Business, Technology, Marketing and Finance, API, OSD, Marketing, Generic drug, Medicinal plants, Risk management

The "Taste Masking and Taste Assessment Services and Technologies Market, Distribution by Type of Formulation, Type of Techniques, Scale of Operation and Key Geographical Regions: Industry Trends and Global Forecasts, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Taste Masking and Taste Assessment Services and Technologies Market, Distribution by Type of Formulation, Type of Techniques, Scale of Operation and Key Geographical Regions: Industry Trends and Global Forecasts, 2022-2035" report has been added to ResearchAndMarkets.com's offering.
  • 'Taste Masking and Taste Assessment Services and Technologies Market, 2022-2035 report features an extensive study of the current market landscape, offering an informed opinion on the taste masking and taste assessment services and technologies.
  • One of the key objectives of the report was to estimate the existing market size and future growth potential of the taste masking and taste assessment services and technologies market.
  • How is the current and future opportunity, related to taste masking and taste assessment likely to be distributed across key market segments?

AgEagle Announces Enhancements to Measure Ground Control, Designed to Support and Manage Autonomous Drone Operations of Any Scale

Retrieved on: 
Friday, May 13, 2022
Azure, API, Economics, Software, Mavic, Ground control, ESRI, Goal, Photogrammetry, Solution, Government, Workflow, Microsoft Power BI, Polydactyly, Phantom, Mooney, Trust, Control, Securities Act of 1933, Securities Exchange Act of 1934, Weather, Salesforce, Industry, Time, Lists of disasters, ArcGIS, AWS, Maritime Transportation Security Act of 2002, QGIS, Adoption, NYSE, CEO, KML, GLOBE, Surveying, Risk management, Medical imaging, Beekeeping, Matrice, Agriculture

Analyze drone data or view orthomosaics and other 2D data files on an interactive, account-wide map.

Key Points: 
  • Analyze drone data or view orthomosaics and other 2D data files on an interactive, account-wide map.
  • Collaborate and support operations with detailed information about missions, including flight logs with screen shots, playbacks and incident flagging; and efficiently manage equipment and workflows with automatic usage tracking capabilities.
  • Benefit from Measure Ground Controls obsession to deliver industry-leading, customer-centric support and service.
  • For sales information on Measure Ground Control or to discuss collaborative technology integration opportunities, please contact [email protected] .

Grand City Properties S.A. announces FY 2021 results

Retrieved on: 
Wednesday, March 16, 2022
EPS, Annual report, AGM, Acquisition, EPRA, Income, Publication, AFRICA, Frankfurt Stock Exchange, GCP, Polydactyly, Environment, ISIN, Company, NTA, CEO, ICR, Growth, Shelf registration, Grand City Properties, FFO, Share repurchase, LTV, Security (finance), Lithium, NRW, Grand duchy

- Portfolio vacancy reached a historic low at 5.1% as of year-end 2021, driven by strong operational performance and asset recycling.

Key Points: 
  • - Portfolio vacancy reached a historic low at 5.1% as of year-end 2021, driven by strong operational performance and asset recycling.
  • - Solid FFO I growth, with FFO I for 2021 of 186 million, up by 2% as compared to 2020.
  • Luxembourg, March 16, 2022 - Grand City Properties S.A. ("GCP" or the "Company") announces results for the financial year 2021.
  • Refael Zamir, CEO of Grand City Properties: "Throughout 2021, we continued our focus on delivering progress across all activities, delivering continued sustainable stakeholder value creation, benefiting tenants, the environment, our local communities, and rewarding our shareholders.

DGAP-News: Grand City Properties S.A. announces FY 2021 results

Retrieved on: 
Wednesday, March 16, 2022
EPS, Annual report, AGM, Acquisition, EPRA, Income, Publication, AFRICA, Frankfurt Stock Exchange, GCP, Polydactyly, Environment, ISIN, Company, NTA, CEO, ICR, Growth, Shelf registration, Grand City Properties, FFO, Share repurchase, LTV, Security (finance), Lithium, NRW, Grand duchy

- Portfolio vacancy reached a historic low at 5.1% as of year-end 2021, driven by strong operational performance and asset recycling.

Key Points: 
  • - Portfolio vacancy reached a historic low at 5.1% as of year-end 2021, driven by strong operational performance and asset recycling.
  • - Solid FFO I growth, with FFO I for 2021 of 186 million, up by 2% as compared to 2020.
  • Luxembourg, March 16, 2022 - Grand City Properties S.A. ("GCP" or the "Company") announces results for the financial year 2021.
  • Refael Zamir, CEO of Grand City Properties: "Throughout 2021, we continued our focus on delivering progress across all activities, delivering continued sustainable stakeholder value creation, benefiting tenants, the environment, our local communities, and rewarding our shareholders.