Fetus

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

Retrieved on: 
Mittwoch, Mai 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

Retrieved on: 
Mittwoch, Mai 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

Retrieved on: 
Mittwoch, 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

Retrieved on: 
Dienstag, 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

Retrieved on: 
Freitag, 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: 
Sonntag, 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.

Announcement Execution of Memorandum of Understanding for Share Acquisition (Subsidiarization)

Retrieved on: 
Mittwoch, April 3, 2024

The Target Company is a company engaged in a prenatal diagnosis business providing genetic testing and analysis services.

Key Points: 
  • The Target Company is a company engaged in a prenatal diagnosis business providing genetic testing and analysis services.
  • We believe that business synergies will be generated as we aim to improve the value of the customer experience.
  • This acquisition is in line with our ongoing strategy of obtaining assets which can contribute to expanding our presence in Japan.
  • Making the Target Company our subsidiary by purchase of shares (cash settlement), share exchange (kabushiki koukan) or other methods, or a combination thereof.

Calliditas Therapeutics to Present Nefecon Data at the ISN World Congress of Nephrology April 13 - 16 in Buenos Aires

Retrieved on: 
Montag, April 8, 2024

The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.

Key Points: 
  • The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.
  • Presentations will highlight the eGFR results found in patients on Nefecon as well as the data on quality of life during the trial.
  • There will also be a presentation on the subanalysis evaluating benefits of Nefecon for patients with lower levels of UPCR.
  • The congress will include a symposium, Evolving Landscape of eFGR and Proteinuria Surrogate Markers in IgA Nephropathy, moderated by KOL Richard Lafayette, M.D., F.A.C.P.

Calliditas Therapeutics to Present Nefecon Data at the ISN World Congress of Nephrology April 13 - 16 in Buenos Aires

Retrieved on: 
Montag, April 8, 2024

The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.

Key Points: 
  • The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.
  • Presentations will highlight the eGFR results found in patients on Nefecon as well as the data on quality of life during the trial.
  • There will also be a presentation on the subanalysis evaluating benefits of Nefecon for patients with lower levels of UPCR.
  • The congress will include a symposium, Evolving Landscape of eFGR and Proteinuria Surrogate Markers in IgA Nephropathy, moderated by KOL Richard Lafayette, M.D., F.A.C.P.

U.S. FDA Approves Broad New Labels for NEXLETOL® and NEXLIZET® to Prevent Heart Attacks and Cardiovascular Procedures in Both Primary and Secondary Prevention Patients, Regardless of Statin Use

Retrieved on: 
Freitag, März 22, 2024

In addition, the enhanced labels support the use of NEXLETOL and NEXLIZET either alone or in combination with statins.

Key Points: 
  • In addition, the enhanced labels support the use of NEXLETOL and NEXLIZET either alone or in combination with statins.
  • They also include new indications for primary hyperlipidemia, alone or in combination with a statin, and are the only LDL-C lowering non-statin drugs indicated for primary prevention patients.
  • NEXLETOL and NEXLIZET are also the first oral non-statin LDL-C lowering drugs to be approved by the FDA to reduce the risk of CV events in both primary and secondary prevention patients.
  • CLEAR Outcomes is part of the CLEAR clinical research program for NEXLETOL® (bempedoic acid) Tablet and NEXLIZET® (bempedoic acid and ezetimibe) Tablet.