Regenxbio's MPS II Gene Therapy RGX-121 Hit With CRL Noah Stansfield

Published Date: 11 Feb 2026

The FDA issued a CRL for gene therapy RGX-121 in Hunter syndrome, citing trial design and biomarker doubts; Regenxbio seeks a meeting with the agency and resubmission.

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NeurologyLive® Friday 5 — February 6, 2026 Isabella Ciccone, MPH

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 6, 2026.

Innovative Trial Employs Novel PET Imaging to Track Cladribine’s Effect on Microglial Activation Louie Pasculli

The single-arm CLAD-MAPET study is evaluating whether cladribine tablets reduce microglial activation, captured by [F-18]PBR06-PET, in patients with MS experiencing progression without relapse or MRI activity on anti-CD20 therapy.

Cladribine Tablets Reduce Treatment Switches, Outpatient Visits, and Health Care Costs in Multiple Sclerosis Isabella Ciccone, MPH

A real-world study showed that cladribine tablets were associated with greater treatment persistence and lower health care costs in patients with multiple sclerosis compared with other common therapies.

Comparative Study Reveals Psychiatric Comorbidities Common in MOGAD, MS, and NMOSD, With Distinct Timing Patterns Isabella Ciccone, MPH

A new comparison study of patients with autoimmune diseases reported high rates of anxiety and depression, with notable differences in when psychiatric symptoms emerged relative to disease onset.

Phase 3b RESHAPE Study Evaluates Risk–Benefit of Switching From Ocrelizumab to BTK Inhibitor Remibrutinib Isabella Ciccone, MPH

A phase 3b study presented at ACTRIMS 2026 is evaluating whether switching to remibrutinib offers efficacy and safety comparable to continued ocrelizumab treatment in relapsing multiple sclerosis.

Myelin Protective Small Molecule Lucid-MS Shows Promising Early-Stage Data in Healthy Volunteers Marco Meglio

Findings from a phase 1 study indicated that Lucid-21-302 was generally well tolerated and exhibited dose-proportional exposure, warranting additional study in a phase 2 trial for multiple sclerosis.

Todays Updates

Ultragenyx Puts BLA for MPS IIIA Gene Therapy UX111 in Front of FDA Again Noah Stansfield

Ultragenyx seeks FDA accelerated approval for UX111 gene therapy in Sanfilippo syndrome, citing neurologic benefit and the use of cerebral spinal fluid heparan sulfate data.

This Week on NeurologyLive® — February 9, 2026 Isabella Ciccone, MPH

Here's some of what is coming soon to NeurologyLive® this week.

AGNOS Study Results Demonstrate Ofatumumab’s Impact on Neurofilament Light, GFAP Marco Meglio

AGNOS trial showed that monthly treatment with ofatumumab lowered NfL and GFAP in patients with early RRMS, signaling reduced nerve damage and bolstering first-line treatment potential.

International Epilepsy Day 2026: Highlighting Gaps in Pediatric Care, Stigma, and Education Charuta Joshi, MD, Isabella Ciccone, MPH

Pediatric neurologist Charuta Joshi, MD, discussed global disparities in epilepsy for children and adolescents amid International Epilepsy Day, highlighting opportunities for education, stigma reduction, and improved care access.

Discussing Billing Challenges in Dermatology, With Daniel Siegel, MD Daniel M. Siegel, MD

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New Phase 3 Findings Support Ameluz-PDT for Patients with Actinic Keratosis

New Phase 3 Findings Support Ameluz-PDT for Patients with Actinic Keratosis

Todays Updates

Ultragenyx Puts BLA for MPS IIIA Gene Therapy UX111 in Front of FDA Again Noah Stansfield

Ultragenyx seeks FDA accelerated approval for UX111 gene therapy in Sanfilippo syndrome, citing neurologic benefit and the use of cerebral spinal fluid heparan sulfate data.

This Week on NeurologyLive® — February 9, 2026 Isabella Ciccone, MPH

Here's some of what is coming soon to NeurologyLive® this week.

AGNOS Study Results Demonstrate Ofatumumab’s Impact on Neurofilament Light, GFAP Marco Meglio

AGNOS trial showed that monthly treatment with ofatumumab lowered NfL and GFAP in patients with early RRMS, signaling reduced nerve damage and bolstering first-line treatment potential.

International Epilepsy Day 2026: Highlighting Gaps in Pediatric Care, Stigma, and Education Charuta Joshi, MD, Isabella Ciccone, MPH

Pediatric neurologist Charuta Joshi, MD, discussed global disparities in epilepsy for children and adolescents amid International Epilepsy Day, highlighting opportunities for education, stigma reduction, and improved care access.

Discussing Billing Challenges in Dermatology, With Daniel Siegel, MD Daniel M. Siegel, MD

In this interview, Siegel highlights key points presented in his Maui Derm 2026 talk, 'How To Maximize Your Billing Effectiveness in the Era of Dwindling Reimbursements and ICD-10.'

New Phase 3 Findings Support Ameluz-PDT for Patients with Actinic Keratosis

New Phase 3 Findings Support Ameluz-PDT for Patients with Actinic Keratosis

Latest News

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 6, 2026.

The single-arm CLAD-MAPET study is evaluating whether cladribine tablets reduce microglial activation, captured by [F-18]PBR06-PET, in patients with MS experiencing progression without relapse or MRI activity on anti-CD20 therapy.

A real-world study showed that cladribine tablets were associated with greater treatment persistence and lower health care costs in patients with multiple sclerosis compared with other common therapies.

A new comparison study of patients with autoimmune diseases reported high rates of anxiety and depression, with notable differences in when psychiatric symptoms emerged relative to disease onset.

A phase 3b study presented at ACTRIMS 2026 is evaluating whether switching to remibrutinib offers efficacy and safety comparable to continued ocrelizumab treatment in relapsing multiple sclerosis.

Findings from a phase 1 study indicated that Lucid-21-302 was generally well tolerated and exhibited dose-proportional exposure, warranting additional study in a phase 2 trial for multiple sclerosis.

News All Time

Women Physicians Day 2026: Prioritizing Whole-Person Care for Women With Movement Disorders Isabella Ciccone, MPH, Jill Farmer, DO, MPH, FCPP, DipABLM, Kelly Papesh, DNP, APRN, FNP-C, CMRD, Madhureeta Achari, MD, Joanna Dearlove, MD

As part of National Women Physicians Day, a group of women clinicians discussed various topics in the care for women with movement disorders.

National Women Physicians Day: Perspectives From Riley Bove, MD Louie Pasculli, Riley Bove, MD

Riley Bove, MD, MS, associate professor of neurology at the University of California San Francisco, shares her personal view on National Women’s Physician’s and why it’s important for clinicians and patients in the field.

National Women Physicians Day: Women Driving Change in Neurology Louie Pasculli

In honor of National Women Physician’s Day, the NeurologyLive® team highlighted recent studies on women transforming neurologic care and advancing women’s health.

National Women Physicians Day: Insights From Aleksandra Pikula, MD, DipABPN, DipABLM, FELMO Aleksandra Pikula, MD, DipABPN, DipABLM, FELMO, Louie Pasculli

Aleksandra Pikula, MD, DipABPN, DipABLM, FELMO, director of the JS Sonshine Center, shares her personal view on National Women Physician’s and why it’s important for clinicians and patients in the field.

VMAT2 Inhibitor NBI-1065890 Heads to Phase 2 Study in Tardive Dyskinesia Marco Meglio

Neurocrine Biosciences launches a Phase 2 trial for NBI-1065890, targeting tardive dyskinesia with promising new treatment options.

FDA Clears KORU FreedomEDGE System to Administer Rozanolixizumab-noli in gMG Louie Pasculli

The FDA approves the FreedomEDGE infusion platform for rozanolixizumab, enhancing treatment options for generalized myasthenia gravis in clinical settings.

NeurologyLive® Brain Games: February 1, 2026 Marco Meglio, Louie Pasculli

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on the ACTRIMS and its history!

Anti-IL6R Therapy Effective in Reducing NMOSD and MOGAD Disease Activity Louie Pasculli

Anti–interleukin-6 receptor therapy may offer a new treatment option for patients with NMOSD and MOGAD who do not respond to rituximab, according to new findings.

This Week on NeurologyLive® — February 2, 2026 Isabella Ciccone, MPH

Here's some of what is coming soon to NeurologyLive® this week.

National Women Physicians Day: Janis Miyasaki, Neuropalliative Care, and Advancing Women Leadersip Marco Meglio, Janis Miyasaki, MD, MEd, FRCPC, FAAN

Janis Miyasaki, MD, MEd, FRCPC, FAAN, a pioneer in neuropalliative care, provided clinical thoughts and perspectives on National Women Physicians Day and the ways to promote women leadership within the neurology field.

Women Physicians Day 2026: Supporting Women With MS Through Reproductive and Life Stage Transitions Isabella Ciccone, MPH, Katrina Bawden, MSN, MSCN, FNP-C, Parisa Khosravi, DO, April Erwin, MD

In honor of National Women Physicians Day, held February 3, 2026, a trio of women clinicians highlighted the importance of individualized care for women living with multiple sclerosis.

National Women Physicians Day: Strengthening the Pathway for Women Physicians in Neuroscience Marco Meglio, Claire Henchcliffe, MD, DPhil

Claire Henchcliffe, MD, DPhil, an international leader in Parkinson disease research, reflects on mentorship, leadership development, and the systemic changes needed to support long-term success for women in neurology and clinical neuroscience.

Women Physicians Day 2026: Sex-Specific Clinical Considerations in Women With Epilepsy Isabella Ciccone, MPH, Page B. Pennell, MD, FAES, FAAN, FANA

Epilepsy titan Page B. Pennell, MD, FAES, FAAN, FANA, outlined key hormonal, reproductive, and treatment-related factors that influence epilepsy management and outcomes for women in different life stages.

NeurologyLive® Friday 5 — January 30, 2026 Isabella Ciccone, MPH

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 30, 2026.

AAN Publishes First-Ever Guideline for Functional Seizure Care Management Marco Meglio

The AAN's new guideline enhances diagnosis and management of functional seizures, emphasizing psychological interventions and addressing care gaps for better patient outcomes.

Taysha Doses First Patient in Pivotal Trial for Rett Syndrome Gene Therapy TSHA-102, Aligns With FDA on BLA Plans Noah Stansfield

Specifically, the alignment relates to a separate, planned clinical trial referred to as ASPIRE.

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