Wolman Disease News
3D miniature livers lead the way to patient-specific drug discovery - Science Daily
Fri, 02 Aug 2019 07:00:00 GMT
3D miniature livers lead the way to patient-specific drug discovery  Science Daily

Researchers have developed a reproducible method for creating multicellular miniature liver organoids from pluripotent stem cells. Steatohepatitis, a liver ...

Lysosomal Acid Lipase Deficiency (LAAL) Treatment Market – Geographic Segmentation and Competitive Landscape, Segmented by End-user – Growth, Trends, and Forecast 2019 – 2024 - Spaceflight News
Sun, 18 Aug 2019 13:18:11 GMT
Lysosomal Acid Lipase Deficiency (LAAL) Treatment Market – Geographic Segmentation and Competitive Landscape, Segmented by End-user – Growth, Trends, and Forecast 2019 – 2024  Spaceflight News

Lysosomal Acid Lipase Deficiency (LAAL) Treatment Market – Geographic Segmentation and Competitive Landscape, Segmented by End-user – Growth, ...

Scientists bioengineer human liver disease in the lab to find new treatments - EurekAlert
Thu, 30 May 2019 07:00:00 GMT
Scientists bioengineer human liver disease in the lab to find new treatments  EurekAlert

Scientists successfully bioengineered human liver organoids that faithfully mimic key features of fatal liver disease in the laboratory. This allowed them to ...

Global Lysosomal Acid Lipase Deficiency Treatment Market 2019 Insights, Share, Growth and Future Trends - Financial Newspaper
Tue, 13 Aug 2019 11:48:04 GMT
Global Lysosomal Acid Lipase Deficiency Treatment Market 2019 Insights, Share, Growth and Future Trends  Financial Newspaper

Lysosomal Acid Lipase Deficiency Treatment market report involves an in-depth understanding of Lysosomal Acid Lipase Deficiency Treatment market and ...

Scientists Bioengineer Human Liver Disease in the Lab - Laboratory Equipment
Fri, 31 May 2019 07:00:00 GMT
Scientists Bioengineer Human Liver Disease in the Lab  Laboratory Equipment

Scientists successfully bioengineered human liver organoids that faithfully mimic key features of fatal liver disease in the laboratory.

Amicus, Thermo Fisher's Brammer Bio Partner on Gene Therapy Manufacturing - Genetic Engineering & Biotechnology News
Tue, 02 Jul 2019 07:00:00 GMT
Amicus, Thermo Fisher's Brammer Bio Partner on Gene Therapy Manufacturing  Genetic Engineering & Biotechnology News

Amicus Therapeutics will address clinical and commercial supply needs for its intrathecal adeno-associated virus (AAV) Batten disease gene therapy programs ...

In-Depth Company Profiles of Key Players, Upcoming Prominent Players Revenue, Gross Margin, and Future Market Prospects Until 2025 of Global Lysosomal Acid Lipase Deficiency Treatment market - Honest Version
Mon, 05 Aug 2019 11:52:27 GMT
In-Depth Company Profiles of Key Players, Upcoming Prominent Players Revenue, Gross Margin, and Future Market Prospects Until 2025 of Global Lysosomal Acid Lipase Deficiency Treatment market  Honest Version

Trusted Business Insights presents the Latest Study on Lysosomal Acid Lipase Deficiency Treatment Market 2019-2025. The report contains market predictions ...

Lysosomal Acid Lipase Deficiency Unmasked in Two Children With Nonalcoholic Fatty Liver Disease - AAP News
Tue, 22 Nov 2016 13:31:24 GMT
Lysosomal Acid Lipase Deficiency Unmasked in Two Children With Nonalcoholic Fatty Liver Disease  AAP News

Lysosomal acid lipase deficiency (LAL-D) is a classic lysosomal storage disorder characterized by accumulation of cholesteryl ester and triglyceride. Although it ...

Amicus Expands into Gene Therapy with Celenex Acquisition - Genetic Engineering & Biotechnology News
Thu, 20 Sep 2018 07:00:00 GMT
Amicus Expands into Gene Therapy with Celenex Acquisition  Genetic Engineering & Biotechnology News

Amicus Therapeutics has acquired Nationwide Children's Hospital spinout Celenex for up to $452 million, in a deal that expands the buyer into gene therapy ...

(2019-2025) Lysosomal Acid Lipase Deficiency (LAAL) Treatment Market Top Companies, Business Insights,Growth, Market Size Global Market Share, Global Market Size, Trends, Sales, Revenue, Forecast and Detailed Analysis - Bіrmіnhаm Nеws
Mon, 22 Jul 2019 07:00:00 GMT
(2019-2025) Lysosomal Acid Lipase Deficiency (LAAL) Treatment Market Top Companies, Business Insights,Growth, Market Size Global Market Share, Global Market Size, Trends, Sales, Revenue, Forecast and Detailed Analysis  Bіrmіnhаm Nеws

Global “Lysosomal Acid Lipase Deficiency (LAAL) Treatment Market” will reach xxx Million USD in 2019 and CAGR xx% 2019-2025. The report begins from ...

Amicus Therapeutics’ Expanded Portfolio Includes Potential Gene Therapies for Batten Disease - Batten Disease News
Fri, 28 Sep 2018 07:00:00 GMT
Amicus Therapeutics’ Expanded Portfolio Includes Potential Gene Therapies for Batten Disease  Batten Disease News

Read more about the novel gene therapies focused on the treatment of Batten disease and other lysosomal storage disorders that Amicus Therapeutics ...

FDA OKs First Drug (Kanuma) for Rare Enzyme Disease - Medscape
Tue, 08 Dec 2015 08:00:00 GMT
FDA OKs First Drug (Kanuma) for Rare Enzyme Disease  Medscape

The US Food and Drug Administration (FDA) today approved sebelipase alfa (Kanuma, Alexion Pharmaceuticals), the first treatment for a rare genetic condition ...

FDA approves drug from transgenic chicken - Nature.com
Thu, 31 Dec 2015 08:00:00 GMT
FDA approves drug from transgenic chicken  Nature.com

The FDA granted a first ever approval to a drug that is produced in the egg white of transgenically engineered chickens. The agency granted the green light to ...

Genetically engineered chickens produce eggs used in new human drug - dvm360
Mon, 14 Dec 2015 08:00:00 GMT
Genetically engineered chickens produce eggs used in new human drug  dvm360

The U.S. Food and Drug Administration (FDA) has approved a new drug called Kanuma (sebelipase alfa) as a treatment for patients with a rare disease called ...

Lysosomal Acid Lipase (LAL) Deficiency Treatment Market – Kanuma, the only approved therapy to reign the market - Truth Daily Mirror
Thu, 28 Feb 2019 08:00:00 GMT
Lysosomal Acid Lipase (LAL) Deficiency Treatment Market – Kanuma, the only approved therapy to reign the market  Truth Daily Mirror

Lysosomal acid lipase (LAL) deficiency is a rare inherited condition in which the body is not able to produce enough lysosomal acid lipase (LAL) enzyme, which ...

Clinical trial enrollment gets harder as patient populations shrink - BioPharma Dive
Mon, 10 Apr 2017 07:00:00 GMT
Clinical trial enrollment gets harder as patient populations shrink  BioPharma Dive

There are currently no cures or treatments directly targeting Berger's disease. The rare illness, which affects about 130,000 people in the U.S. annually, is the ...

GMO Chicken: FDA Approves Genetically Modified Hens, But For Medicine, Not Food - International Business Times
Thu, 10 Dec 2015 08:00:00 GMT
GMO Chicken: FDA Approves Genetically Modified Hens, But For Medicine, Not Food  International Business Times

The U.S. Food and Drug administration has approved a genetically modified chicken, but not the variety that Americans will have to worry about finding in the ...

The price of a miracle: Should we limit spending on lifesaving drugs? - The Conversation US
Wed, 05 Jul 2017 07:00:00 GMT
The price of a miracle: Should we limit spending on lifesaving drugs?  The Conversation US

Specialty prescription drugs are responsible for countless medical miracles, but their high price tag is the main reason health care costs are out of control.

How Much Should We Spend on Miracle Drugs? (Op-Ed) - Live Science
Fri, 07 Jul 2017 07:00:00 GMT
How Much Should We Spend on Miracle Drugs? (Op-Ed)  Live Science

This article was originally published at The Conversation. The publication contributed the article to Live Science's Expert Voices: Op-Ed & Insights. "It was the ...

Synageva reports good data for cholesterol-lowering drug - Boston Business Journal
Wed, 04 Sep 2013 07:00:00 GMT
Synageva reports good data for cholesterol-lowering drug  Boston Business Journal

Synageva BioPharma Corp. (Nasdaq: GEVA) says that patients who have been on its potential treatment for lysosomal acid lipase (LAL) deficiency for one year ...


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