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Top News In Lysosomal Today - Breaking & Trending Today

What is Cholesteryl Ester Storage Disease?

Cholesteryl Ester Storage Disease (CESD) is a rare autosomal recessive multisystem condition that belongs to the lysosomal storage disorders family. ....

Marko Aliaksandr Shutterstock , Sophia Coveney , Therapeutic Development , Cholesteryl Ester Storage Disease , Cholesteryl Ester Storage , Image Credit , Lysosomal Acid Lipase , Lysosomal Storage , Clinical Cases , Diagnostic Algorithm , Clinical Presentation , Pediatric Gastroenterology , Lancet Gastroenterology , Diagnostic Pathology , Targeting Wolman Disease , Disease Pathogenesis , Current Chemical Genomics , Translational Medicine ,

CENTOGENE Extends Strategic Partnership With Takeda to Continue Providing Access to Genetic Testing for Patients With Lysosomal Storage Disorders

The global market access and expansion partnership, initially established in 2015, aims to provide an efficient and timely diagnosis to patients with Lysosomal Storage Disorders Diagnosis plays a. | April 11, 2023 ....

United Kingdom , Ian Rentsch , Ben Legg , Lennart Streibel , Corporate Communications , Shire Pharmaceuticals , Drug Administration , Exchange Commission , Lysosomal Storage Disorders , Commercial Officer , General Manager , Insight Reports , Centogenenv Stock Exchange , Press Release , Lays Cntg Nl0014040206 ,

Centogene Extends Genetic Testing Partnership With Takeda

By Chris Wack

Centogene N.V. said Tuesday that it has extended its partnership with Takeda Pharmaceutical Co. to diagnose patients with Lysosomal Storage Disorders.

Under the renewed. | April 11, 2023 ....

Chris Wack , Shire Pharmaceuticals , Takeda Pharmaceutical Co , Takeda Pharmaceutical , Lysosomal Storage , Dow Jones , Centogenenv Stock Exchange , Press Release , He Cntg Nl0014040206 ,

Partnership to investigate Lysosomal Modulators

The Medicines Discovery Institute, Cardiff University (MDI) and Astex Pharmaceuticals, a Cambridge-based developer of small molecule therapeutics for oncology and diseases of the central nervous system, are working together to identify new drugs to treat neurodegenerative diseases with high unmet medical need. Click to read more. ....

United Kingdom , David Rees , Helen Waller Evans , Astex Pharmaceuticals , Medicines Discovery Institute , Cardiff University , Simon Ward , Chief Scientific Officer , Medicines Discovery ,

Passage Bio Receives European Commission Orphan Designation for PBKR03 for Treatment of Krabbe Disease

- Urgency for effective treatment underscored by European Commission designation for investigational gene therapy PBKR03
- Global Phase 1/2 trial – GALax-C – PBKR03 planned to initiate in first half of 2021
PHILADELPHIA, April 05, 2021 (GLOBE NEWSWIRE) Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the European Commission has granted Orphan designation for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). The designation was based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. Currently, there are no approved disease-modifying therapies available for Krabbe disease, a rare lysosomal storage disease that most often presents early in a child’s life. The U.S. Food and Drug Administrat ....

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