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Rare Disease
10 Articles Available
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Rare Diseases
Staying Agile to Bring Therapies to Patients with Rare Diseases
PAO-06-022-CL-08
Rare Disease Clinical Trials
Rare Disease Patients Benefit from Clinical Trial Flexibility
PAO-02-022-CL-03
Rare Diseases
Leveraging AI and RNA to Fight Rare Diseases
PAO-02-22-R250-17
Gene Therapy
Beyond 2020: Looking into the Crystal Ball for Gene Therapy
PAO-01-21-CL-03
Endocrine Diseases
Developing Transformative Treatments for Rare Endocrine Diseases
PAP-Q2-20-CL-023
Drug Approval
U.S. FDA Approves VYNDAQEL® and VYNDAMAX™ for Use in Patients with Transthyretin Amyloid Cardiomyopathy, a Rare and Fatal Disease
PR-M05-19-NI-036
Clinical Trial Logistics
Facilitating Complex Clinical Trials For Rare Diseases
PAP-Q1-2019-CL-003
Cell and Gene Therapy
REGENXBIO and Abeona Therapeutics Announce Worldwide Exclusive Licenses for the Treatment of Four Rare Lysosomal Storage Disorders Using NAV AAV9 Vector
PR-M11-18-NI-028
Drug Development
Patients with Rare Leukemia Disease Will Have New Treatment Option
PAO-M09-18-NI-016
Business
Expansion Therapeutics Raises $55.3 Million in Series A Financing to Advance Portfolio of Novel RNA Targeted Small Molecule Medicines to Treat Rare Diseases
PR-M01-18-NI-009
