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Critical Path Institute and Centogene N.V. are pleased to announce the signing of a Memorandum of Understanding to advance drug development for lysosomal diseases and improve the quality of life for those impacted by these conditions.

JCR Pharmaceuticals’ Research Presentations at WORLD Symposium ™ 2024 Showcase JR-141 (Pabinafusp Alfa) and Other Investigational Treatments for Lysosomal Storage Disorders.

Denali Therapeutics Inc. (Nasdaq: DNLI) today announced program progress and expected milestones for 2024, which Chief Executive Officer, Ryan Watts, Ph.D., will highlight during a corporate presentation at the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday, January 9th, at 10:30 a.m. Pacific Time.

Arkuda Therapeutics today announced that it entered into an option and asset purchase agreement with Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company, which grants Janssen an exclusive option to purchase Arkuda’s portfolio of lysosomal function enhancers.

M6P Therapeutics, a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders, announced the presentation of promising preclinical data at the 19th Annual WORLDSymposium™, a research conference dedicated to lysosomal diseases being held in Orlando, FL, and virtually Feb. 22-26, 2023.

We are in an unprecedented time in neurotherapeutics. Medicines that address the causative disease biology underlying central nervous system

Amicus Therapeutics (Nasdaq: FOLD), today announced that WORLD Symposium™ has awarded Pombiliti™ ( cipaglucosidase alfa-atga ) + Opfolda™ ( miglustat ) the 2024 New Treatment Award, which recognizes important achievements in advancing treatments for lysosomal diseases which have attained regulatory approval.

AVROBIO to Present Clinical and Preclinical Data from Lysosomal Disorder Gene Therapy Pipeline at WORLD Symposium ™ 2023.

Centogene N.V., the essential life science partner for data-driven answers in rare and neurodegenerative diseases, announced it has extended its partnership with Takeda to diagnose patients with Lysosomal Storage Disorders.

Gain Therapeutics, Inc. today announced the publication of preclinical data identifying a novel class of small molecule allosteric regulators that demonstrate therapeutic potential for galactosidase beta 1 (GLB1)-related lysosomal storage disorders (LSDs), including GM1 gangliosidosis.