UCLA-led gene therapy approved by FDA for rare, life-threatening immune disorder

A groundbreaking gene therapy developed through a UCLA-led clinical trial has received approval from the U.S. Food and Drug ...

Rocket’s gene therapy Kresladi wins FDA approval in rare immune disease

Upon approval, Rocket has been awarded a priority review voucher, which currently fetch around $200m on the secondary market.

ACD AWARDS FELLOWSHIP GRANTS TO ADVANCE CREATINE DEFICIENCY RESEARCH AT SIX RESEARCH CENTERS

Six 2026 ACD Fellowship Grantees will spend a year conducting research to advance treatment options for Cerebral ...

FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder

Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug ...
Verywell Health on MSN

Researcher shows anxiety could be linked to a nutritional deficiency

Medically reviewed by Patricia Mikula, PharmD Key Takeaways Choline is an essential nutrient needed to make acetylcholine, a ...
WTEN

Malta family spreading awareness about rare genetic disorder

Rian McCann is almost seven years old. Her parents, Danielle and Frank described her as kind, sassy, smart and above all, resilient. Rian was born with CDKL5 Deficiency Disorder — which prevents her ...
BioPharma Dive

Rocket gene therapy cleared by FDA for rare immune disorder

Though the therapy, Kresladi, isn’t seen as a big seller, its clearance is a step forward for a company that’s lost most of ...
Nature

Sulfite Oxidase Deficiency and Related Metabolic Disorders

Sulfite oxidase deficiency is a rare, inherited neurometabolic disorder arising from impaired detoxification of sulfite, a by-product of sulfur‐containing amino acid catabolism. The condition is most ...
clinicaladvisor.com

Vitamin D Deficiency Linked to Neurologic Disorders

Thus, treating vitamin D deficiency in patients with neurocognitive disorders to an endpoint of 50 ng/mL may be considered. 1 Dana S. Miles, MS, PA-C, CAQ-EM, has been working in emergency medicine as ...
Nature

Beta-Ketothiolase Deficiency and Related Metabolic Disorders

Beta-ketothiolase deficiency is a rare autosomal recessive disorder that disrupts both ketone body utilisation and isoleucine catabolism. Caused by mutations in genes such as ACAT1, which encodes ...
Seeking Alpha

European Commission approval of Ztalmy® (ganaxolone) for the adjunctive treatment of epileptic seizures associated with CDKL5 deficiency disorder

The European Commission has granted marketing authorisation in the European Union (EU) for Ztalmy ® (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with ...
CMAJ

Irreversible blindness induced by vitamin A deficiency in a child with avoidant/restrictive food intake disorder secondary to Noonan syndrome

Vitamin A deficiency should be considered in children with unexplained vision loss, particularly those with restrictive eating disorders such as avoidant/restrictive food intake disorder. Early ...