Rare Disease Day, observed annually on the last day of February, serves as an international campaign to raise awareness about the challenges faced by individuals and families living with rare diseases ...
Please provide your email address to receive an email when new articles are posted on . An Italian pharmaceutical company and a Boston-area biotechnology research firm announced a partnership to ...
On the occasion of this Rare Disease Day, Mstone Partners Healthcare and our portfolio companies are proud to stand with the ...
December 2, 2011 — Lysosomal storage disorders such as Fabry's disease and Pompe's disease are much more common than previously thought, particularly atypical later-onset forms, a new study suggests.
Ahmedabad: The city recently got the national biobank for lysosomal storage disorders at the FRIGE Institute of Human Genetics. Lysosomal storage disorders (LSDs) are a diverse group of over 70 rare, ...
Azafaros Announces Publication of Preclinical Efficacy Data with Nizubaglustat in GM2 Gangliosidosis
Azafaros, a company aiming to become a leader in lysosomal storage disorders (LSDs), focused on addressing especially neurological symptoms, today announced the publication of proof-of-concept ...
HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today ...
Analysis from continued follow-up of Phase 1/2 clinical trial data in Hunter syndrome (MPS II) reinforces potential for tividenofusp alfa ...
In the past week, two announcements from the drug discovery world have had Insilico Medicine’s generative artificial intelligence (AI) target discovery engine, PandaOmics, front and center. The first ...
LONDON--(BUSINESS WIRE)--Technavio’s latest market research report on the global lysosomal storage diseases (LSDs) market provides an analysis of the most important trends expected to impact the ...
For Zevra Therapeutics and its rare neurodegenerative disease med arimoclomol, the second time’s the charm. Friday, the FDA approved Zevra’s arimoclomol capsules to treat the rare lysosomal storage ...
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