Fetus

First-In-Class Cholesterol-Lowering Treatment NILEMDO® (NEXLETOL® in the U.S.) and Its Combination with Ezetimibe, NUSTENDI® (NEXLIZET® in the U.S.), Approved In Europe To Treat Hypercholesterolemia and Significantly Reduce Cardiovascular Risk

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onsdag, maj 22, 2024

ANN ARBOR, Mich. and MUNICH, Germany, May 22, 2024 (GLOBE NEWSWIRE) -- Daiichi Sankyo Europe GmbH (hereafter, ‘Daiichi Sankyo’) and Esperion Therapeutics, Inc. jointly announced today that the European Commission (EC) has approved the label update of both NILEMDO® (bempedoic acid) and NUSTENDI® (bempedoic acid / ezetimibe fixed-dose combination (FDC)), as treatments for hypercholesterolemia (high levels of cholesterol) and to reduce the risk of adverse cardiovascular events. The EC’s decisions to update the labels of bempedoic acid and bempedoic acid / ezetimibe FDC are based on the positive CLEAR Outcomes trial results and makes them the first and only LDL-C lowering treatments indicated for primary and secondary prevention of cardiovascular events.

Key Points: 
  • Bempedoic acid is a first-in-class oral treatment which lowers cholesterol, and which can be combined with other treatments to help lower cholesterol even further.
  • Bempedoic acid provided additional cholesterol lowering of up to 28% on top of statin therapy, compared to placebo.
  • “Today's announcement marks a pivotal moment in our ongoing efforts to reduce cardiovascular risk.
  • CLEAR Outcomes is part of the CLEAR clinical research program for NEXLETOL® (bempedoic acid) Tablet and NEXLIZET® (bempedoic acid and ezetimibe) Tablet.

NATERA ALERT: Bragar Eagel & Squire, P.C. is Investigating Natera, Inc. on Behalf of Long-Term Stockholders and Encourages Investors to Contact the Firm

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onsdag, maj 15, 2024

Our investigation concerns whether the board of directors of Natera have breached their fiduciary duties to the company.

Key Points: 
  • Our investigation concerns whether the board of directors of Natera have breached their fiduciary duties to the company.
  • Natera, a Delaware corporation with principal executive offices in Austin, Texas, offers genetic testing in the areas of women’s health, oncology, and organ health.
  • Among other things, The New York Times reported that Natera’s positive results for several genetic disorders were incorrect more than 80 percent of the time.
  • In addition, the FDA advised health care providers that they should not rely on NIPT results alone to diagnose chromosomal abnormalities or disorders.

Children’s Hospital Los Angeles Forms Nursing Leader Roundtable to Improve Best Practices at Regional Neonatal Intensive Care Units

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fredag, maj 10, 2024

Children’s Hospital Los Angeles ( CHLA ) has created the innovative Regional NICU Nursing Leadership Roundtable, comprised of CHLA and 20 community hospital neonatal intensive care units (NICU) in Los Angeles, Ventura and Kern counties.

Key Points: 
  • Children’s Hospital Los Angeles ( CHLA ) has created the innovative Regional NICU Nursing Leadership Roundtable, comprised of CHLA and 20 community hospital neonatal intensive care units (NICU) in Los Angeles, Ventura and Kern counties.
  • The initiative was developed by Kathy Van Allen, MSN, RN, CPN, the Administrative Director of CHLA’s Fetal and Neonatal Institute .
  • View the full release here: https://www.businesswire.com/news/home/20240510231203/en/
    Kathy Van Allen, administrative director of the Fetal and Neonatal Institute at Children's Hospital Los Angeles.
  • “The roundtable provides a forum to discuss critical issues impacting NICU nursing practice and to create a supportive network for the NICU leaders,” says Van Allen.

Emma - The Sleep Company's Bedtime Story to Strengthen Bonds Harnessing the Power of Maternal Voice for Working Mothers on Mother's Day

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fredag, maj 10, 2024

Neuroscience research has shown that mother's voice leaves a distinct 'neural fingerprint' that can predict a child's proficiency in social communication.

Key Points: 
  • Neuroscience research has shown that mother's voice leaves a distinct 'neural fingerprint' that can predict a child's proficiency in social communication.
  • Emma – The Sleep Company proposes a way for the children of working mothers to feel their mothers' presence, even when their mothers can't be physically there.
  • NEW YORK, May 10, 2024 /PRNewswire/ -- This year, for Mother's Day, we want to put the focus on working mothers.
  • From the womb, a fetus begins to recognise and favour its mother's voice, and this preference continues after birth.

New US product, ORMALVI™ (dichlorphenamide) tablets, launches for Primary Periodic Paralysis (PPP) treatment

Retrieved on: 
onsdag, maj 8, 2024

We want to provide them with the support that they require to effectively manage their PPP.

Key Points: 
  • We want to provide them with the support that they require to effectively manage their PPP.
  • ORMALVI™ is an oral carbonic anhydrase inhibitor indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants.
  • Simply Stated: Research Updates in Periodic Paralysis - Quest | Muscular Dystrophy Association [online] Available at: https://mdaquest.org/simply-stated-research-updates-in-periodic-paralysis Accessed 23 Nov. 2023.
  • The impact of permanent muscle weakness on quality of life in periodic paralysis: a survey of 66 patients.

Natera Launches Fetal RhD NIPT Supporting Ob/Gyn Physicians and Patients During RhIg Shortage

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onsdag, maj 1, 2024

Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA (cfDNA) and genetic testing, today announced the launch of a new cfDNA-based fetal RhD test.

Key Points: 
  • Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA (cfDNA) and genetic testing, today announced the launch of a new cfDNA-based fetal RhD test.
  • This comes at a critical time for the healthcare industry, helping physicians navigate patient care given nationwide shortages of Rho(D) immune globulin therapy (RhIg).
  • Natera’s test can be performed as early as nine weeks gestation and determines fetal RhD status from the blood of a pregnant patient, including complex pseudogene and RhD-CE-D hybrid variants.
  • The vast majority of other NIPT laboratories do not offer fetal RhD assessment, which makes it a key differentiator in addition to Natera’s core SNP-based technology.

BILLIONTOONE MITIGATING RHOGAM(R) SHORTAGE WITH UNITY FETAL RhD™ NON-INVASIVE PRENATAL TEST

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onsdag, april 17, 2024

MENLO PARK, Calif., April 17, 2024 /PRNewswire/ -- BillionToOne, a molecular diagnostics company with a mission to create powerful and accurate tests that are accessible to all, has reached a milestone of testing over 80,000 patients for Rh incompatibility, which develops when a pregnant patient has Rh-negative blood and the baby has Rh-positive blood. In rare cases this incompatibility can lead to a serious issue in which the pregnant patient's immune system will attack their baby's red blood cells as foreign.

Key Points: 
  • However, the FDA recently announced that the most common brand of this medication, RhoGAM(R), is in shortage .
  • Utilizing cell-free DNA in maternal plasma, the UNITY Fetal RhD Non-Invasive Prenatal Test can determine fetal RhD status," said Jennifer Hoskovec, MS, Certified Genetic Counselor and Senior Director of Medical Affairs at BillionToOne.
  • The UNITY Fetal RhD NIPT offers a reliable and convenient alternative to traditional methods of determining fetal RhD status, such as paternal testing and invasive procedures like amniocentesis.
  • The UNITY Fetal RhD NIPT accurately determines the RhD status of the fetus from maternal blood as early as 10 weeks gestation.

AMGEN TO PRESENT TEZSPIRE® PHASE 2A COPD DATA AT ATS 2024

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tisdag, april 16, 2024

THOUSAND OAKS, Calif., April 16, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today provided an update regarding the results of the Phase 2a COURSE trial for TEZSPIRE® (tezepelumab-ekko) in chronic obstructive pulmonary disease (COPD), which were accepted for presentation in the Clinical Trials Symposium at The American Thoracic Society (ATS) 2024 International Conference on Monday, May 20, from 9:15-11:15 a.m. PDT. 

Key Points: 
  • Overall, tezepelumab numerically reduced the annualized rate of moderate or severe COPD exacerbations versus placebo by 17% (90% CI: −6, 36; p=0.1042).
  • Of note, more reductions were observed in a prespecified subgroup of patients with BEC ≥150 cells/μL (37% [95% CI: 7, 57]).
  • The trend in reduction was greater in a small number of subjects with BEC ≥300 cells/µL.
  • We look forward to presenting the full data set at the congress and based on these findings, we are actively planning for the Phase 3 development of tezepelumab in COPD.

Type 2 diabetes is not one-size-fits-all: Subtypes affect complications and treatment options

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fredag, april 19, 2024

You may have heard of Ozempic, the “miracle drug” for weight loss, but did you know that it was actually designed as a new treatment to manage diabetes?

Key Points: 
  • You may have heard of Ozempic, the “miracle drug” for weight loss, but did you know that it was actually designed as a new treatment to manage diabetes?
  • In Canada, diabetes affects approximately 10 per cent of the general population.

Locks and keys

  • Every cell in the body needs sugar as an energy source, but too much sugar can be toxic to cells.
  • This equilibrium needs to be tightly controlled and is regulated by a lock and key system.
  • Cells cover themselves with locks that respond perfectly to insulin keys to facilitate the entry of sugar into cells.
  • The body can encounter difficulties producing an adequate number of insulin keys, and/or the locks can become stubborn and unresponsive to insulin.

Severe insulin-deficient diabetes: We’re missing keys!

  • In the severe insulin-deficient diabetes (SIDD) subtype, the key factories — the beta cells — are on strike.
  • Why the beta cells go on strike remains largely unknown, but since there is an insulin deficiency, treatment often involves insulin injections.

Severe insulin-resistant diabetes: But it’s always locked!

  • In the severe insulin-resistant diabetes (SIRD) subtype, the locks are overstimulated and start ignoring the keys.
  • There are many treatment avenues for these patients but no consensus about the optimal approach; patients often require high doses of insulin.

Mild obesity-related diabetes: The locks are sticky!

  • Mild obesity-related (MOD) diabetes represents a nuanced aspect of Type 2 diabetes, often observed in individuals with higher body weight.
  • The locks are “sticky,” so it is challenging for the key to click in place and open the lock.

Mild age-related diabetes: I’m tired of controlling blood sugar!


Mild age-related diabetes (MARD) happens more often in older people and typically starts later in life. With time, the key factory is not as productive, and the locks become stubborn. People with MARD find it tricky to manage their blood sugar, but it usually doesn’t lead to severe complications. Among the different subtypes of diabetes, MARD is the most common.

Unique locks, varied keys

  • In Canada, unique cases of Type 2 diabetes were identified in Indigenous children from Northern Manitoba and Northwestern Ontario by Dr. Heather Dean and colleagues in the 1980s and 90s.
  • Read more:
    Indigenous community research partnerships can help address health inequities

    Childhood-onset Type 2 diabetes is on the rise across Canada, but disproportionately affects Indigenous youth.

  • Acknowledging this distinct subtype of Type 2 diabetes in First Nations communities has led to the implementation of a community-based health action plan aimed at addressing the unique challenges faced by Indigenous Peoples.

A mosaic of conditions

  • Type 2 diabetes is not uniform; it’s a mosaic of conditions, each with its own characteristics.
  • Since diabetes presents so uniquely in every patient, even categorizing into subtypes does not guarantee how the disease will evolve.


Lili Grieco-St-Pierre receives funding from Fonds de recherche du Québec - Santé (FRQS). Jennifer Bruin receives funding from the Canadian Institutes of Health Research (CIHR), Natural Sciences and Engineering Research Council of Canada (NSERC), JDRF, Diabetes Canada.

Esperion Presents Important New Data from CLEAR Outcomes at ACC.24 Highlighting Value of NEXLETOL® (bempedoic acid) Tablets in Diverse Populations Including Women, Hispanics/Latinx and Patients with Obesity

Retrieved on: 
söndag, april 7, 2024

ANN ARBOR, Mich., April 07, 2024 (GLOBE NEWSWIRE) -- Esperion (Nasdaq: ESPR) today announced the presentation of results from three pre-specified subgroups from CLEAR Outcomes at the 2024 American College of Cardiology’s Annual Scientific Sessions (ACC.24): women, Hispanic/Latinx, and patients with obesity. These results align with the American College of Cardiology’s robust diversity, equity and inclusion programs to drive cultural change across the profession and ensure that the cardiovascular care team is as diverse as the patients they care for and that all patients are represented in cardiovascular research. The data also reinforce the mission of the ACC: transforming cardiovascular care for all.

Key Points: 
  • “Bempedoic acid is the only FDA approved non-statin LDL lowering therapy to demonstrate reductions in MACE in both primary prevention and secondary prevention patient populations.
  • The Hispanic population is the largest ethnic minority in the U.S., yet is a population historically underrepresented in clinical trials.
  • At Esperion, we discover, develop, and commercialize innovative medicines to help improve outcomes for patients with or at risk for cardiovascular and cardiometabolic diseases.
  • CLEAR Outcomes is part of the CLEAR clinical research program for NEXLETOL® (bempedoic acid) Tablet and NEXLIZET® (bempedoic acid and ezetimibe) Tablet.