FDA Approves First Treatment for Stem Cell Transplant–Associated Thrombotic Microangiopathy

The U.S. Food and Drug Administration (FDA) has approved Yartemlea (narsoplimab‑wuug), marking the first authorized therapy for hematopoietic stem cell transplant‑associated thrombotic microangiopathy (TA‑TMA) in adults and children aged 2…

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Biohaven’s BHV‑7000 Falls Short in Midstage Depression Trial, Refocusing Pipeline Strategy

As reported on FierceBioTech, Biohaven closed out the holiday period with disappointing news: its investigational Kv7.2/7.3 potassium‑channel modulator, BHV‑7000, did not demonstrate efficacy in a phase 2 clinical trial for…

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It Was All a Dream

Editor's Note: Patient Worthy is honored to share Michelle Patidar's patient story, provided to us by our friends at Elephants & Tea. To see the article in its original format,…

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From Cancer Treatment to Childhood Health: How Foresee’s Injectable Drug Addresses a Rare Pediatric Challenge

When pharmaceutical companies develop medications for serious diseases like prostate cancer, few anticipate how those treatments might transform care for entirely different patient populations. Yet, according to FiercePharma.com, Foresee Pharmaceuticals…

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CRISPR Therapeutics Shares Promising Updates on Zugo-cel in Autoimmune and Oncology Trials

CRISPR Therapeutics has announced encouraging early results for zugocaptagene geleucel (zugo-cel), its investigational allogeneic CAR T therapy targeting CD19, in both autoimmune diseases and hematologic malignancies. Autoimmune Disease Progress In…

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Chiesi Group Partners with Aliada Therapeutics to Advance Blood-Brain Barrier Technology for Rare Diseases

Chiesi Group has entered an exclusive licensing agreement with Aliada Therapeutics, a subsidiary of AbbVie, to develop therapies that can cross the blood-brain barrier (BBB) for lysosomal storage disorders (LSDs).…

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