Quoin Reports Clinically Meaningful Whole Body Response to QRX003 in Pediatric Netherton Syndrome; Second Patient Cleared for Dosing
Initial pediatric subject shows sustained benefit with QRX003; second patient approved for full-body treatment in ongoing ...
Christian Daily International on MSN1d
Global orphan care network to host free month-long online training to strengthen Christian response for vulnerable children
World Without Orphans (WWO), a global movement dedicated to ensuring every child grows up in a safe, loving family, has ...
Organization in need of donations due to increase in foster care
WILLIAMSPORT, Pa. — Bins of children's clothing can be found inside of Dwell Orphan Care's Hope Chest Foster Closet in ...
Targeted Oncology17h
FDA Oncology Actions: March 2025 Approvals and Designations
On March 5, 2025, the FDA cleared an investigational new drug application for CTD402, a CD7-targeted universal CAR T-cell ...
Exousia Pro Issues and Update on its FDA Orphan Drug Status
ORLANDO, FL / ACCESS Newswire / April 1, 2025 / MARIJUANA INC. (OTC PINK:MAJI), dba Exousia Pro, Inc., a clinical-stage biotech company using exosomes in the treatment of cancer and other maladies, is ...
Fierce Biotech16h
Atsena raises $150M for ocular gene therapies with Bain, Sofinnova in tow
The $150 million will also go toward Atsena’s preclinical pipeline and expand the use of the biotech’s spreading AAV.SPR ...
CURE1d
First Patient Dosed With Annamycin and Cytarabine in Phase 3 AML Trial
The first patient with relapsed or refractory acute myeloid leukemia has been dosed with Annamycin plus Cytarabine in the ...
Natalia Grace: The story of orphan abandoned for being ‘adult’, who inspired Hulu show ‘Good American Family’
Hulu’s new series ‘Good American Family’ is generating a lot of buzz. It’s based on the life of Natalia Grace, the Ukrainian ...
The Manila Times1d
Pharvaris Announces Orphan Designation Granted to Deucrictibant by the European Commission
The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation to deucrictibant for the treatment of bradykinin-mediated angioedema in March 2022.
CURE2d
Relacorilant Plus Chemo Meets Primary Goal in Ovarian Cancer Trial
The phase 3 trial showed relacorilant with chemotherapy improved progression-free and overall survival in platinum-resistant ...