CAR-T Trial Raises Prospect of Preventing Myeloma in High-Risk Group

Published Date: 12 May 2026

All 20 smoldering myeloma patients had deep responses after one-time infusion

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Todays Updates


Evaluating Zeleciment Basivarsen in Myotonic Dystrophy Type 1: The Phase 3 HARMONIA Trial Isabella Ciccone, MPH


The phase 3 HARMONIA trial will evaluate whether a muscle- and CNS-targeted antisense conjugate improves function across multiple systems in patients with myotonic dystrophy type 1.

Zilucoplan Autoinjector Matches Bioequivalence of Pre-Filled Syringe in Myasthenia Gravis Marco Meglio


New data from the 2026 AANEM Annual Meeting showed zilucoplan delivered via autoinjector was bioequivalent to the pre-filled syringe and was well tolerated when self-administered by patients with generalized myasthenia gravis.

Global Phase 3 UPSTREAM MG Trial of Telitacicept Advances Enrollment in Generalized Myasthenia Gravis Isabella Ciccone, MPH


The ongoing phase 3 study is enrolling approximately 180 adults across more than 13 countries, with topline results expected in mid-2027.

EpilepsyGTx, Viralgen Partner on AAV Gene Therapy EPY201 Ahead of Planned First-in-Human Trial Louie Pasculli


EpilepsyGTx and Viralgen have entered a manufacturing collaboration to support development of EPY201, an investigational AAV9-based gene therapy for focal refractory epilepsy planned to enter first-in-human testing in 2027.

New Phase 3 Trials to Test Empasiprubart Against Placebo, IVIG in CIDP Louie Pasculli


Phase 3 trials EMNERGIZE and EMVIGORATE will evaluate the efficacy and safety of intravenous empasiprubart in adults with chronic inflammatory demyelinating polyradiculoneuropathy.

NeuroVoices: James Winkley, MD, on Analyzing Phase 3 PREVAIL Trial of Gefurulimab Marco Meglio


The chairman of the Neurology Service Line at Baptist Health in Kentucky discussed PREVIEW trial findings for gefelorilimab in generalized myasthenia gravis, including efficacy, safety, and what the subcutaneous C5 inhibitor could offer patients if approved.

Lessons From 10,000 Organ Transplants, With Ari Cohen, MD


Lessons From 10,000 Organ Transplants, With Ari Cohen, MD

Todays Updates


Evaluating Zeleciment Basivarsen in Myotonic Dystrophy Type 1: The Phase 3 HARMONIA Trial Isabella Ciccone, MPH


The phase 3 HARMONIA trial will evaluate whether a muscle- and CNS-targeted antisense conjugate improves function across multiple systems in patients with myotonic dystrophy type 1.

Zilucoplan Autoinjector Matches Bioequivalence of Pre-Filled Syringe in Myasthenia Gravis Marco Meglio


New data from the 2026 AANEM Annual Meeting showed zilucoplan delivered via autoinjector was bioequivalent to the pre-filled syringe and was well tolerated when self-administered by patients with generalized myasthenia gravis.

Global Phase 3 UPSTREAM MG Trial of Telitacicept Advances Enrollment in Generalized Myasthenia Gravis Isabella Ciccone, MPH


The ongoing phase 3 study is enrolling approximately 180 adults across more than 13 countries, with topline results expected in mid-2027.

EpilepsyGTx, Viralgen Partner on AAV Gene Therapy EPY201 Ahead of Planned First-in-Human Trial Louie Pasculli


EpilepsyGTx and Viralgen have entered a manufacturing collaboration to support development of EPY201, an investigational AAV9-based gene therapy for focal refractory epilepsy planned to enter first-in-human testing in 2027.

New Phase 3 Trials to Test Empasiprubart Against Placebo, IVIG in CIDP Louie Pasculli


Phase 3 trials EMNERGIZE and EMVIGORATE will evaluate the efficacy and safety of intravenous empasiprubart in adults with chronic inflammatory demyelinating polyradiculoneuropathy.

NeuroVoices: James Winkley, MD, on Analyzing Phase 3 PREVAIL Trial of Gefurulimab Marco Meglio


The chairman of the Neurology Service Line at Baptist Health in Kentucky discussed PREVIEW trial findings for gefelorilimab in generalized myasthenia gravis, including efficacy, safety, and what the subcutaneous C5 inhibitor could offer patients if approved.

Lessons From 10,000 Organ Transplants, With Ari Cohen, MD


Lessons From 10,000 Organ Transplants, With Ari Cohen, MD

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