MyChesCo on MSN4h
AscellaHealth Launches Specialty & Rare Pipeline Digest to Guide Pharmaceutical Stakeholders
AscellaHealth has introduced its latest Specialty & Rare Pipeline Digest™, a quarterly publication designed to provide ...
Louisiana families fight rare condition
Louisiana families are fighting for awareness and recognition of a rare neurological condition that affects children.
Fierce Pharma9h
For struggling cell and gene therapy field, Peter Marks' FDA exit adds even more uncertainty
The sudden resignation of Peter Marks, M.D., Ph.D., from the FDA branch that regulates biological products for humans ...
Man with high levels of pregnancy hormone is diagnosed with 'exceptionally rare' cancer
The unnamed 52-year-old man did not know he had the disease and he originally visited a doctor complaining of a painless swelling in the lower right side of his abdomen.
The American Journal of Managed Care12h
Areas of Unmet Need Continue to Burden Patients With gMG
Patient-reported outcomes measures in generalized myasthenia gravis (gMG) are more important than ever, for both those ...
BioSpace13h
Denali Goes All in On Neuroscience’s Thorniest Hurdle: Crossing the Blood-Brain Barrier
After some high-profile crashes, the one-time biotech darling is inching toward success with its Hunter syndrome treatment, ...
Expensive but lifesaving testing for cancer, Alzheimers to be covered by insurance in N.J.
Biomarker testing also extends beyond cancer care. The science is being used in the treatment of other diseases including ...
The Manila Times14h
AscellaHealth Releases Q1 2025 Specialty & Rare Pipeline Digest™ Providing Essential Clinical Drug Updates
The FDA's focus on specialty medications and orphan drugs continues to expand, with 71% of recent submissions being for ...
'I thought I had a sinus infection - then doctors told me I have rare incurable disease'
After thinking he just had a stubborn sinus infection that wouldn't go away, Brian McNamara was told he had an ultra-rare ...
EXCLUSIVE: How 1 dad used AI to advance finding a treatment for his son’s rare disease
AI helped Thomas Wagner understand Alexander disease, find connection with other conditions and discover researchers who ...
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 ...