Aug 12, 2020
Immune Thrombocytopenia (ITP) Market To Reach USD 3.68 Billion By 2027 | Reports And Data
Jun 18, 2020
FDA Approves First Therapy for Rare Disease that Causes Low Phosphate Blood Levels, Bone Softening
Jun 16, 2020
FDA Approves First Treatment for Adult Onset Still's Disease, a Severe and Rare Disease
Jun 12, 2020
EHA25Virtual: Pegcetacoplan, a Novel C3 Complement Inhibitor - Beginning of a New Era in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Jun 11, 2020
Rigel to Present Poster Highlighting TAVALISSE at the Virtual Edition of the 25th European Hematology Association (EHA) Annual Congress
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
Jun 01, 2020
PTC Therapeutics Successfully Completes Acquisition of Censa Pharmaceuticals
May 28, 2020
Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Submission of Biologics License Application to U.S. Food and Drug Administration for Pegunigalsidase Alfa for the Treatment of Fabry Disease
May 27, 2020
Immune Thrombocytopenia Market is Anticipated to Grow by 2030 Owing to a Rich and Robust Pipeline
May 19, 2020
Eiger BioPharmaceuticals Announces FDA Acceptance of NDA for Filing with Priority Review of Zokinvy(TM) (lonafarnib) for Treatment of Progeria and Progeroid Laminopathies
May 19, 2020
NORD Awards Eleven New Grants in Eight Disease States for Rare Disease Research
May 14, 2020
Paroxysmal Nocturnal Hemoglobinuria Markets and Pipeline Analysis: 2016-2019 & 2020-2024
May 14, 2020
Akcea Announces Approval for Reimbursement of TEGSEDI® (inotersen) in Spain for Treatment of Hereditary Transthyretin Amyloidosis with Polyneuropathy
May 11, 2020
BioSpecifics Reports First Quarter 2020 Financial and Operating Results
May 11, 2020
Protalix BioTherapeutics Announces Positive Topline Results from the BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease
May 08, 2020
Synlogic Reports First Quarter 2020 Financial Results and Provides Business Update
May 06, 2020
PTC Therapeutics to Acquire Censa Pharmaceuticals
May 05, 2020
Rigel Reports First Quarter 2020 Financial Results and Provides Business Update
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 30, 2020
InMed Receives CTA Approval for Second Phase 1 Clinical Trial with INM-755