Jun 01, 2020
CytRx Corporation Highlights Orphazyme's Rolling Submission of its New Drug Application with the U.S. FDA for Arimoclomol in Niemann-Pick Disease Type-C
May 03, 2020
BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement to Develop Gene Therapies for Rare Genetic Cardiomyopathies
Apr 29, 2020
CytRx Corporation Highlights Updated Arimoclomol Milestone Guidance Provided by Orphazyme A/S in Niemann-Pick Disease
Apr 08, 2020
Polaryx Therapeutics Receives IND Approval From the FDA to Study PLX-200 Treatment for Patients With Juvenile Neuronal Ceroid Lipofuscinosis
Mar 12, 2020
Metachromatic Leukodystrophy (MLD) Market Assessment, 2020 - Pipeline Products, Unmet Needs, Market Drivers & Barriers, Competitive Landscape
Mar 12, 2020
Protalix BioTherapeutics Announces $43.7 Million in Financing to Further Advance its Programs in Fabry Disease
Feb 11, 2020
CytRx Corporation Highlights Recently Published Data of its Licensee Orphazyme A/S Phase 2 Trial of Arimoclomol in the Treatment of Sporadic Inclusion Body Myositis (sIBM)
Feb 10, 2020
Takeda to Present Research Advances in Rare Lysosomal Storage Disorders at 16th Annual WORLDSymposium(TM) 2020
Feb 06, 2020
Protalix BioTherapeutics and Chiesi Farmaceutici Announce Receipt of "Agreement Letter" for Initial Pediatric Study Plan for PRX-102 for the Treatment of Fabry Disease
Feb 04, 2020
CytRx Corporation Highlights Updated Arimoclomol Milestone Guidance Provided by Licensee Orphazyme A/S
Jan 29, 2020
REGENXBIO Announces Presentations at the 16th Annual WORLDSymposium(TM) 2020
Jan 20, 2020
Polaryx Therapeutics Receives IND Approval for PLX-200 From the FDA for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis
Dec 23, 2019
Polaryx Therapeutics Submits Investigational New Drug Application to the U.S. FDA for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis With PLX-200
Dec 19, 2019
CytRx Corporation Highlights Significant Positive Events From its Two Licensed Drugs Arimoclomol and Aldoxorubicin
Oct 22, 2019
Global Tay-Sachs Disease Pipeline Insights 2019 by Discovery and Preclinical, Phase 1, Phase 2, and Phase 3 Stage
Sep 24, 2019
Protalix BioTherapeutics and Chiesi Group Complete Enrollment in the Third Phase III Clinical Trial of pegunigalsidase alfa (PRX-102) for the Treatment of Fabry Disease
Aug 28, 2019
Niemann-Pick Disease Type C (NPC) Insights, Epidemiology, and Market Forecasts, 2028
Jul 30, 2019
CytRx Corporation Highlights Arimoclomol Licensee Orphazyme A/S Prepares for Regulatory Filing of Arimoclomol in U.S. for Niemann-Pick Disease Type C
Jul 22, 2019
CytRx Corporation Highlights Arimoclomol Licensee Orphazyme A/S Completes Enrollment in its Phase 3 Clinical Trial of Arimoclomol in Amyotrophic Lateral Sclerosis
May 30, 2019
Scientists Bioengineer Human Liver Disease in the Lab to Find New Treatments