Pediatric Rare Diseases Archives

LogicBio cleared to continue dosing in pediatric MMA trial

Acid Disorders, Clinical Hold, Clinical Trials, FDA, Gene Mutations, Methylmalonic Acidemia (MMA), Pediatric Rare Diseases, R&D, Rare Diseases, Severe adverse event (SAE)

LogicBio Therapeutics was granted the green light to continue dosing participants in the company’s Phase I/II Sunrise trial investigating the safety of LB-001 in patients with methylmalonic acidemia (MMA), characterized by methylmalonyl-CoA mutase (MMUT) gene mutations.

Takeda touts positive pediatric data in competitive HAE space

Clinical Trials, Hereditary Angioedema (HAE), Pediatric, Pediatric Diseases, Pediatric Rare Diseases, R&D, Takeda, Therapeutics

Takeda Pharmaceutical announced positive results from the Phase III SHP643-301 trial of Takhzyro (lanadelumab) in kids ages 2 to <12 years with hereditary angioedema (HAE). The drug is already approved for HAE in people 12 years and older.

Sumitomo Dainippon Pharma and Roivant Sciences Create Broad Strategic Alliance for New Medicines

Alliances, Biopharmaceutical, Business, Drug Distribution, Market Access, Market exclusivity, New Medicines, Pediatric Rare Diseases, Prostate Cancer, Respiratory Disease, Stakes, Urinary Diseases, Women’s Health

The Sumitomo Dainippon-Roivant Alliance encompasses up to 11 biopharmaceutical Vants with more than 25 innovative clinical programs and multiple potential product launches from 2020 to 2022, and access to key elements of Roivant’s proprietary technology platforms including DrugOme and Digital Innovation.