Autosomal dominant polycystic kidney disease

Regulus Therapeutics to Present Additional Data from its Autosomal Dominant Polycystic Kidney Disease (ADPKD) Program at PKD Connect 2021

Retrieved on: 
Monday, June 21, 2021
Kidney diseases, Medical specialties, Autosomal dominant polycystic kidney disease, Channelopathies, Clinical medicine, Branches of biology, Kidney, Polycystic kidney disease

"This year's conference is an opportune occasion to share with the PKD community our additional data that further validate miR-17 as a target for ADPKD.

Key Points: 
  • "This year's conference is an opportune occasion to share with the PKD community our additional data that further validate miR-17 as a target for ADPKD.
  • The Phase 1b is an adaptive design, open-label, multiple dose study in up to three cohorts of patients with ADPKD.
  • Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.
  • These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission.

Sanofi provides update on venglustat clinical program

Retrieved on: 
Tuesday, June 1, 2021
Rare diseases, Medical specialties, Lipid storage disorders, Clinical medicine, Kidney diseases, Health, Autosomal dominant polycystic kidney disease, Channelopathies, Polycystic kidney disease, Gangliosidosis, GM2 gangliosidoses, Sanofi

PARIS JUNE 1, 2021 A pivotal Phase 2/3 study of venglustat in autosomal dominant polycystic kidney disease (ADPKD) did not meet futility criteria, and the company has halted the clinical program in ADPKD.

Key Points: 
  • PARIS JUNE 1, 2021 A pivotal Phase 2/3 study of venglustat in autosomal dominant polycystic kidney disease (ADPKD) did not meet futility criteria, and the company has halted the clinical program in ADPKD.
  • Sanofi has both completed and active studies evaluating venglustat in Gaucher diseasetype 3, Fabry disease and GM2 Gangliosidosis.
  • Venglustat is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
  • With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.

Reata Announces FDA Accepted for Filing the NDA for Bardoxolone for the Treatment of Patients With Chronic Kidney Disease Caused by Alport Syndrome

Retrieved on: 
Monday, April 26, 2021
Kidney diseases, Medical specialties, Clinical medicine, Medicine, Alport syndrome, Autosomal dominant polycystic kidney disease, Kidney disease, Polycystic kidney disease, Kidney, Dialysis, Polycystic disease

\xe2\x80\x9cAlport syndrome is one of the most rapidly progressive forms of CKD and a truly devastating disease to those patients and the families who are affected by it.

Key Points: 
  • \xe2\x80\x9cAlport syndrome is one of the most rapidly progressive forms of CKD and a truly devastating disease to those patients and the families who are affected by it.
  • The kidneys of patients with Alport syndrome progressively lose the capacity to filter waste products out of the blood, which can lead to end-stage kidney disease and the need for chronic dialysis treatment or a kidney transplant.
  • According to the Alport Syndrome Foundation, Alport syndrome affects approximately 30,000 to 60,000 people in the United States.
  • The FDA has granted Orphan Drug designation to bardoxolone for the treatment of Alport syndrome and autosomal dominant polycystic kidney disease (\xe2\x80\x9cADPKD\xe2\x80\x9d).

Eloxx Pharmaceuticals to Report Fourth Quarter and Full Year 2020 Financial Results and Provide Business Update on March 11, 2021

Retrieved on: 
Thursday, March 4, 2021
Branches of biology, Biology, Molecular biology, Kidney diseases, Gene expression, Molecular genetics, Protein biosynthesis, Genetic code, Polycystic kidney disease, Gene, Point mutation, Autosomal dominant polycystic kidney disease

Live Webcast: accessible from the Company's website at www.eloxxpharma.com under Events and Presentations or with this link: https://edge.media-server.com/mmc/p/m65hdyjn .

Key Points: 
  • Live Webcast: accessible from the Company's website at www.eloxxpharma.com under Events and Presentations or with this link: https://edge.media-server.com/mmc/p/m65hdyjn .
  • Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.
  • Eloxxs lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins.
  • Eloxx also has preclinical programs focused on kidney diseases including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.

Eloxx Announces Publication of Scientific Manuscript on ELX-02 in the Journal of Cystic Fibrosis

Retrieved on: 
Tuesday, February 9, 2021
Medical specialties, Kidney diseases, Clinical medicine, Channelopathies, Rare diseases, Autosomal dominant disorders, Ciliopathy, Autosomal dominant polycystic kidney disease, Cystic fibrosis, Genetic disorder, Polycystic kidney disease, Genetic heterogeneity

These data support our current Phase 2 clinical trial for ELX-02 in cystic fibrosis patients with G542X nonsense alleles, for whom there are few, if any, treatment options available.

Key Points: 
  • These data support our current Phase 2 clinical trial for ELX-02 in cystic fibrosis patients with G542X nonsense alleles, for whom there are few, if any, treatment options available.
  • ELX-02 is in the early stages of clinical development focusing on cystic fibrosis.
  • Eloxx also has preclinical programs focused on kidney diseases including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.
  • Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control.

End Stage Renal Disease Market Size Worth $193.8 Billion By 2028: Grand View Research, Inc.

Retrieved on: 
Wednesday, January 27, 2021
Kidney diseases, Anatomy, Nephrology, Organ failure, Branches of biology, Artificial kidney, Membrane technology, Kidney failure, Chronic kidney disease, Kidney, Autosomal dominant polycystic kidney disease, Renal angina

SAN FRANCISCO, Jan. 27, 2021 /PRNewswire/ -- The global end stage renal disease market size is anticipated to reach USD 193.8 billion by 2028, according to a new report by Grand View Research, Inc.

Key Points: 
  • SAN FRANCISCO, Jan. 27, 2021 /PRNewswire/ -- The global end stage renal disease market size is anticipated to reach USD 193.8 billion by 2028, according to a new report by Grand View Research, Inc.
  • The increasing number of patients suffering from end stage renal disease (also known as kidney failure) and the rising prevalence of hypertension and diabetes are fueling the market growth.
  • Technological advancements including the innovation of wearable artificial kidney (WAK) in the treatment of renal failure are expediting the overall market growth.
  • Grand View Research has segmented the global end stage renal disease market on the basis of treatment and region:
    End-Stage Renal Disease Treatment Outlook (Revenue, USD Million, 2016 - 2028)

End Stage Renal Disease Market Size Worth $193.8 Billion By 2028: Grand View Research, Inc.

Retrieved on: 
Wednesday, January 27, 2021
Kidney diseases, Anatomy, Nephrology, Organ failure, Branches of biology, Artificial kidney, Membrane technology, Kidney failure, Chronic kidney disease, Kidney, Autosomal dominant polycystic kidney disease, Renal angina

SAN FRANCISCO, Jan. 27, 2021 /PRNewswire/ -- The global end stage renal disease market size is anticipated to reach USD 193.8 billion by 2028, according to a new report by Grand View Research, Inc.

Key Points: 
  • SAN FRANCISCO, Jan. 27, 2021 /PRNewswire/ -- The global end stage renal disease market size is anticipated to reach USD 193.8 billion by 2028, according to a new report by Grand View Research, Inc.
  • The increasing number of patients suffering from end stage renal disease (also known as kidney failure) and the rising prevalence of hypertension and diabetes are fueling the market growth.
  • Technological advancements including the innovation of wearable artificial kidney (WAK) in the treatment of renal failure are expediting the overall market growth.
  • Grand View Research has segmented the global end stage renal disease market on the basis of treatment and region:
    End-Stage Renal Disease Treatment Outlook (Revenue, USD Million, 2016 - 2028)

Eloxx Announces Publication of Scientific Manuscript on ELX-02 in the Journal of Clinical Pharmacology

Retrieved on: 
Wednesday, January 20, 2021
Kidney diseases, Medical specialties, Clinical medicine, Polycystic kidney disease, Autosomal dominant polycystic kidney disease, Forward-looking statement, Polycystic disease

Eloxxs lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins.

Key Points: 
  • Eloxxs lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins.
  • Eloxx also has preclinical programs focused on kidney diseases including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.
  • Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions.
  • Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control.

Global Polycystic Kidney Disease Clinical Trial Pipeline Report 2021 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, January 20, 2021
General Health, Health, Clinical trials, Health sciences, Kidney diseases, Medical specialties, Health, Drug discovery, Pharmaceutical industry, Clinical research, Polycystic disease, Clinical trial, Polycystic kidney disease, Kidney, Autosomal dominant polycystic kidney disease

The "Global Polycystic Kidney Disease Clinical Trial Pipeline Highlights - 2021" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Polycystic Kidney Disease Clinical Trial Pipeline Highlights - 2021" report has been added to ResearchAndMarkets.com's offering.
  • Polycystic Kidney Disease Pipeline Highlights - 2021 provides the most up-to-date information on key pipeline products in the global Polycystic Kidney Disease market.
  • It covers emerging therapies for Polycystic Kidney Disease in active clinical development stages including early and late stage clinical trials.
  • The report provides Polycystic Kidney Disease pipeline products by clinical trial stages including both early and late stage development - phase 3 clinical trials, phase 2 clinical trials, phase 1 clinical trials, preclinical research, and discovery stage.

Regulus Therapeutics Collaborates With Bio-Techne For Biomarker Development

Retrieved on: 
Wednesday, December 2, 2020
Branches of biology, Clinical medicine, Medicine, Kidney diseases, Biomarkers, Regulus Therapeutics, Vought, Autosomal dominant polycystic kidney disease, Polycystic kidney disease, SSM-N-8 Regulus, PC1, Exosome

MINNEAPOLIS, Dec. 2, 2020 /PRNewswire/ --Bio-Techne Corporation (NASDAQ: TECH) and Regulus Therapeutics (NASDAQ: RGLS) announced a collaboration to support Regulus with biomarker analysis for the clinical development of RGLS4326, an Autosomal Dominant Polycystic Kidney Disease (ADPKD) treatment.

Key Points: 
  • MINNEAPOLIS, Dec. 2, 2020 /PRNewswire/ --Bio-Techne Corporation (NASDAQ: TECH) and Regulus Therapeutics (NASDAQ: RGLS) announced a collaboration to support Regulus with biomarker analysis for the clinical development of RGLS4326, an Autosomal Dominant Polycystic Kidney Disease (ADPKD) treatment.
  • Regulus has utilized two Bio-Techne branded product lines, ProteinSimple and Exosome Diagnostics, to aid in their clinical trial patient testing.
  • Regulus has elected to collaborate with Bio-Techne for the development of high-performance biomarker assays to support the assessment of PC1 and PC2 levels using the fully automated Simple Western instrument as a read-out.
  • "Regulus is pleased to enter this collaboration with Bio-Techne," said Jay Hagan, Chief Executive Officer of Regulus.