BioWorld. Link to homepage.
BioWorld
BioWorld MedTech
BioWorld Asia
BioWorld Science
Data Snapshots
BioWorld
BioWorld MedTech
Infographics: Dynamic digital data analysis
Special reports
Aging
Alzheimer's disease
Artificial intelligence
Biosimilars
China CAR T
China's GLP-1 landscape
Coronavirus
Infographics: Dynamic digital data analysis
Israel
IVDs on the rise
PFA re-energizes afib market
Radiopharmaceuticals
Rise of obesity
Top Biopharma Trends of 2023
Top Med-tech Trends of 2023
Top Preclinical Trends of 2023
Premium reports
BioWorld Financings Reports
Disease Incidence & Prevalence Summaries
BioWorld. Link to homepage.
sign in
Sign Out
My Account
Subscribe
BioWorld - Monday, October 7, 2024
Breaking News: BioWorld celebrates nine wins at 2024 APEX Awards
See today's BioWorld Science
Home
» Papillon’s PPL-002 awarded US orphan drug designation for Danon disease
X
Upgrade your daily dose of biopharma and medtech news
Subscribe to BioWorld™ news services
See subscription options
To read the full story,
subscribe
or
sign in
.
Endocrine/metabolic
Papillon’s PPL-002 awarded US orphan drug designation for Danon disease
Oct. 2, 2024
No Comments
The FDA has awarded orphan drug designation to Papillon Therapeutics Inc.’s PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, for the treatment of Danon disease.
BioWorld Science
Regulatory
Endocrine/metabolic
Cell therapy
FDA
Orphan drug