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News
Rare diseases
Apr 29, 2020
Jazz Pharmaceuticals Stops Enrollment in Phase 3 Study Evaluating Defibrotide for the Prevention of Veno-Occlusive Disease
Apr 29, 2020
CytRx Corporation Highlights Updated Arimoclomol Milestone Guidance Provided by Orphazyme A/S in Niemann-Pick Disease
Apr 27, 2020
Eiger BioPharmaceuticals Announces EMA Validation of Lonafarnib MAA for Treatment of Progeria and Progeroid Laminopathies
Apr 08, 2020
Polaryx Therapeutics Receives IND Approval From the FDA to Study PLX-200 Treatment for Patients With Juvenile Neuronal Ceroid Lipofuscinosis
Apr 06, 2020
BioMarin Plans Regulatory Submissions for Marketing Authorization of Vosoritide to Treat Children with Achondroplasia in 3Q 2020 in both US and Europe
Apr 01, 2020
InMed Announces Completion of Initial Phase 1 Clinical Trial of INM-755 CBN Cream in Healthy Subjects
Mar 24, 2020
InMed Submits Clinical Trial Application to Evaluate INM-755 in Second Phase 1 Trial
Mar 23, 2020
Lamellar Ichthyosis (LI) Industry Outlook, 2030 Featuring Emerging Drugs TMB-001, Trifarotene (CD5789), and KB105
Mar 23, 2020
Eiger BioPharmaceuticals Completes Submission of New Drug Application to FDA for Lonafarnib for Treatment of Progeria and Progeroid Laminopathies
Mar 20, 2020
InMed Provides Update on Operations in Response to the COVID-19 Global Outbreak
Mar 18, 2020
Pediatrician and Geneticist Dr. Harvey Levy Receives 2020 David L. Rimoin Lifetime Achievement Award in Medical Genetics from the ACMG Foundation for Genetic and Genomic Medicine
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
Mar 10, 2020
InMed Pharmaceuticals Announces Completed Enrollment in Phase 1 Clinical Trial of INM-755 CBN Cream in Healthy Subjects
Feb 27, 2020
Chiesi Global Rare Diseases Recognizes Rare Disease Day and Highlights Plans to Expand Focus on Patient Advocacy in 2020
Feb 12, 2020
Prevalence of Neuromyelitis Optica Spectrum Disorders, Worldwide, 2019-2029
Feb 12, 2020
Global Bell's Palsy Incidence Assessment by Ethnicity & Geography, 2019-2029
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
Protalix BioTherapeutics Presents Key Clinical Data of Pegunigalsidase Alfa for the Treatment of Fabry Disease at the 16th Annual WORLDSymposium(TM) 2020
Feb 10, 2020
Takeda to Present Research Advances in Rare Lysosomal Storage Disorders at 16th Annual WORLDSymposium(TM) 2020
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