Cholestasis facies

MULTIMEDIA UPDATE - Albireo Announces FDA Approval of Bylvay™ (odevixibat), the First Drug Treatment for Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

Retrieved on:

Tuesday, July 20, 2021

Medical specialties, Hepatology, Progressive familial intrahepatic cholestasis, PFIC, Cholestasis, Primary biliary cholangitis, Odevixibat, Liver, Itch, Cholestatic pruritus, Cholestasis facies

– Only once-daily drug indicated for the treatment of pruritus in PFIC –

Key Points:

Albireo is launching Bylvay immediately to accelerate availability for the patients and families impacted by PFIC.
There are an estimated 100,000 patients with cholestatic liver disease without an approved drug treatment.
Bylvay is the first drug treatment approved in the U.S. for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC).
Progressive familial intrahepatic cholestasis (PFIC) is a rare disorder that causes progressive, life-threatening liver disease.

Albireo Announces FDA Approval of Bylvay™ (odevixibat), the First Drug Treatment for Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

Retrieved on:

Tuesday, July 20, 2021

Medical specialties, Hepatology, Progressive familial intrahepatic cholestasis, PFIC, Cholestasis, Primary biliary cholangitis, Odevixibat, Liver, Itch, Cholestatic pruritus, Cholestasis facies

– Only once-daily drug indicated for the treatment of pruritus in PFIC –

Key Points:

Albireo is launching Bylvay immediately to accelerate availability for the patients and families impacted by PFIC.
There are an estimated 100,000 patients with cholestatic liver disease without an approved drug treatment.
Bylvay is the first drug treatment approved in the U.S. for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC).
Progressive familial intrahepatic cholestasis (PFIC) is a rare disorder that causes progressive, life-threatening liver disease.

Mirum Pharmaceuticals Receives Notice of Allowance for Methods of Use Patent for Maralixibat in Alagille Syndrome

Retrieved on:

Monday, April 26, 2021

b'Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a patent application No.

Key Points:

b'Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a patent application No.
The allowed patent application covers methods of treating Alagille syndrome using maralixibat, an investigational treatment being evaluated for patients with Alagille syndrome, a rare and life-threatening cholestatic liver disease.
The majority of patent applications that receive a notice of allowance will proceed to issue as a U.S. patent.
More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

Mirum Pharmaceuticals Honors Alagille Syndrome Awareness Day with Launch of Disease Awareness Campaign

Retrieved on:

Sunday, January 24, 2021

Infectious diseases, Health, Science, Pharmaceutical, Research, Medical specialties, Digestive diseases, Clinical medicine, Hepatology, Alagille syndrome, Ciliopathy, Syndromes affecting the heart, Primary biliary cholangitis, Cholestasis facies, JAG1, Alagille syndrome, Mirum

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide.

Key Points:

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide.
This disease awareness campaign has the potential to help healthcare providers recognize Alagille syndrome in their patients in order to properly address this rare disease, said Roberta Smith, president of the Alagille syndrome Alliance and parent of a child with ALGS.
As we celebrate International Alagille Syndrome Awareness Day, we will focus on sharing Alagille syndrome family stories that emphasize the critical need for continued research and treatment options for patients worldwide.
Alagille syndrome Awareness Day January 24, 2021
Mirum is also proud to champion the Alagille syndrome Alliance and their ongoing work to support ALGS families through education, outreach, and community.

A4250 Market Report 2020-2030 - Coverage of the Late-stage Emerging Therapies (Phase II) for Alagille Syndrome - ResearchAndMarkets.com

Retrieved on:

Friday, December 18, 2020

Health, Other Health, Medical specialties, Digestive diseases, Hepatology, Clinical medicine, Alagille syndrome, Progressive familial intrahepatic cholestasis, Cholestasis, Biliary atresia, Primary biliary cholangitis, Liver, Cholestasis facies

"A4250- Emerging Insight and Market Forecast - 2030" the report provides comprehensive insights about an investigational product for Alagille syndrome in 7 Major Markets.

Key Points:

"A4250- Emerging Insight and Market Forecast - 2030" the report provides comprehensive insights about an investigational product for Alagille syndrome in 7 Major Markets.
Further, it also consists of future market assessments inclusive of the market forecast, SWOT analysis, market competitors, and other emerging therapies.
Albireo is developing odevixibat to treat rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis, biliary atresia and Alagille syndrome.
Other emerging products for Alagille syndrome are giving market competition to A4250 and launch of late-stage emerging therapies in the near future will significantly impact the market.

2020 Alagille Syndrome Epidemiology Forecast to 2030 - ResearchAndMarkets.com

Retrieved on:

Monday, August 17, 2020

Biotechnology, General Health, Pharmaceutical, Health, Medical specialties, Clinical medicine, Hepatology, Organ systems, Syndromes affecting the heart, Alagille syndrome, Ciliopathy, RTT, Cholestasis facies

The disease epidemiology covered in the report provides historical as well as forecasted Alagille syndrome symptoms epidemiology segmented as the Total Prevalent cases of Alagille syndrome, Gender-specific cases of Alagille syndrome, Age-specific cases of Alagille syndrome.

Key Points:

The disease epidemiology covered in the report provides historical as well as forecasted Alagille syndrome symptoms epidemiology segmented as the Total Prevalent cases of Alagille syndrome, Gender-specific cases of Alagille syndrome, Age-specific cases of Alagille syndrome.
The report provides the segmentation of the Alagille syndrome epidemiology by Prevalent Cases of Alagille syndrome in 7MM.
The report provides the segmentation of the Alagille syndrome epidemiology by Gender-specific Prevalent Cases of Alagille syndrome in 7MM.
The report provides the segmentation of the Alagille syndrome epidemiology by Age-specific Prevalent Cases of Alagille syndrome in 7MM.

Mirum’s Maralixibat Demonstrates Transplant-Free Survival for Pediatric Patients with Progressive Familial Intrahepatic Cholestasis

Retrieved on:

Tuesday, June 2, 2020

Biotechnology, Pharmaceutical, Health, Clinical trials, Medical specialties, Digestive diseases, Hepatology, Medicine, Progressive familial intrahepatic cholestasis, PFIC, Alagille syndrome, Cholestasis, Liver, Primary biliary cholangitis, Cholestasis facies, the Maralixibat Phase 2 INDIGO Study, Maralixibat, Mirum Pharmaceuticals

Patients with an sBA response continue to be treated with maralixibat for more than five years with improvements demonstrated across multiple parameters.

Key Points:

Patients with an sBA response continue to be treated with maralixibat for more than five years with improvements demonstrated across multiple parameters.
Maralixibat is a novel, minimally-absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases for pediatric populations.
More than 1,500 individuals have received maralixibat, including more than 100 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).
The companys lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

Durability of Treatment Effect of Mirum Pharmaceuticals’ Maralixibat for Children With Alagille Syndrome Featured in Late Breaking Session at the Liver Meeting 2019

Retrieved on:

Monday, October 21, 2019

In ICONIC, long-term treatment with maralixibat was associated with durability of treatment response on pruritus measures as well as serum bile acids and xanthomas.

Key Points:

In ICONIC, long-term treatment with maralixibat was associated with durability of treatment response on pruritus measures as well as serum bile acids and xanthomas.
Over the course of four years maralixibat was generally well tolerated, and led to control of cholestasis and pruritus in children with Alagille syndrome.
Title: Durability of Treatment Effect with Long-Term Maralixibat in Children with Alagille Syndrome: 4-Year Safety and Efficacy Results from the ICONIC Study
Maralixibat, a minimally absorbed apical sodium-dependent bile acid transporter inhibitor, is in development for treatment of severe cholestatic diseases.
ICONIC is a randomized, controlled long-term phase 2b study of maralixibat in children with Alagille syndrome.

Company Profile for Mirum Pharmaceuticals

Retrieved on:

Thursday, September 26, 2019

Biotechnology, Health, Pharmaceutical, Clinical trials, Medical specialties, Digestive diseases, Hepatology, Medicine, Progressive familial intrahepatic cholestasis, PFIC, Alagille syndrome, Cholestasis, Liver, Cholestasis facies

Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.

Key Points:

Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
The companys lead product candidate, maralixibat, is an investigational oral drug in development for progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS).

Mirum Pharmaceuticals to Present Phase 2 Clinical Results of Maralixibat in Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis at the International Liver Congress

Retrieved on:

Wednesday, March 27, 2019

Medical specialties, Hepatology, Digestive diseases, Medicine, Progressive familial intrahepatic cholestasis, Cholestasis, Alagille syndrome, PFIC, Bile acid, Liver, Intrahepatic cholestasis of pregnancy, Cholestasis facies

Title: Growth analysis in children with progressive familial intrahepatic cholestasis treated with the apical sodium-dependent bile acid transporter inhibitor, maralixibat.

Key Points:

Title: Growth analysis in children with progressive familial intrahepatic cholestasis treated with the apical sodium-dependent bile acid transporter inhibitor, maralixibat.
Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), thereby preventing bile acids from accumulating in the liver.
Maralixibat is being developed as an investigational treatment for rare pediatric liver disorders such as Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).
The company's lead product candidate, maralixibat, is a Phase 3-ready investigational oral drug being evaluated in Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).