Explore REGENXBIO's RGX-121 gene therapy for Hunter syndrome, with an FDA decision due Nov 9, 2025. Click here to read my most recent analysis.

The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for the treatment of mucopolysaccharidosis II ...

Denali Therapeutics DNLI reported a first-quarter 2025 loss of 78 cents per share, wider than the Zacks Consensus Estimate of a loss of 71 cents. The company reported a loss of 68 cents in the ...

Although middle child syndrome has made its way into current-day lingo, Dr. Welsh cautions that there isn’t enough “robust data” to fully back up the ideas of birth order theory.

Recognizing and detecting the signs that commonly precede sudden arrhythmic death syndrome (SADS) may help to prevent premature deaths, according to research presented today at ESC Preventive ...

Shares of Denali Therapeutics DNLI gained 11.8% on Wednesday after the company announced that it has initiated a rolling submission of a biologics license application (BLA) for accelerated ...

Meanwhile, H.C. Wainwright adjusted its price target for Denali to $80.00, down from $87.00, while maintaining a Buy rating, citing operational expenses and market penetration projections for DNL310.

Early warning signs of Cushing’s syndrome: Excess weight around belly (central obesity) Rounded or puffy face (moon face) Excess hair growth on face, neck, chest, abdomen, and thighs (hirsutism) ...

Denali eyes approval after Hunter syndrome drug clears safety measures, reduces biomarkers in open-label study By Darren Incorvaia Feb 6, 2025 12:05pm ...

GC Biopharma announced on the 5th that the Ministry of Food and Drug Safety has designated the treatment for severe Hunter syndrome, 'Hunterase ICV (intracerebroventricular),' as an orphan drug.

Hunter syndrome results from the absence of an enzyme needed to break down cellular waste. Without it, waste builds up in the body, causing progressive damage to various systems.