MDS Congress 2026: Late-Breaking Data Snapshot Marco Meglio

Published Date: 17 Oct 2026

A quick-read roundup of 10 late-breaking abstracts from the 2026 MDS Congress, from phase 3 Parkinson disease readouts to long-term data in Huntington disease and Tourette syndrome, with the key results that matter for practice.

Read Full News

Latest News

Dompé Begins Phase 2 Trial Testing Intranasal Nerve Growth Factor for Cerebral Palsy Marco Meglio

The Galassia trial will evaluate the safety and tolerability of intranasal cenegermin in about 60 children with spastic cerebral palsy across Italy.

ELEVIDYS Shows Sustained Functional Differences vs External Controls in Older Ambulatory Duchenne Patients Louie Pasculli

New analyses found differences in functional outcomes over 2 years among patients aged 8 to 12 years treated with delandistrogene moxeparvovec, while separate findings characterized micro-dystrophin expression in children younger than 3 years.

Fenfluramine Shows Consistent Seizure Reduction Across Age Groups in CDKL5 Deficiency Disorder Isabella Ciccone, MPH

An analysis of the phase 3 GEMZ trial presented at CNS 2026 showed that fenfluramine produced comparable seizure reductions and safety outcomes in children and adults with CDKL5 deficiency disorder.

Arimoclomol Plus Miglustat Shows Early, Sustained Slowing of Niemann-Pick Disease Type C Progression Isabella Ciccone, MPH

A post hoc analysis of the phase 2/3 NPC002 trial presented at CNS 2026 showed that the benefit of arimoclomol plus miglustat in Niemann-Pick disease type C emerged as early as 3 months after treatment initiation.

NeuroVoices: Ali Habib, MD, on Cemdisiran and the Phase 3 NIMBLE Trial Data in Myasthenia Gravis Marco Meglio, Ali Habib, MD

The professor of neurology and program director of Neuromuscular Medicine at UC Irvine, discusses phase 3 NIMBLE trial findings for cemdisiran in generalized myasthenia gravis, including its novel siRNA mechanism, efficacy, and hospitalization data.

Telitacicept Shows Deepening, Durable Response in Myasthenia Gravis Marco Meglio

New 48-week phase 3 data show many patients with generalized myasthenia gravis reached minimal symptom expression on telitacicept, and most who did sustained it.

Todays Updates

Clinical Forum Weighs in: Biologic Selection in Asthma and COPD

Clinical Forum Weighs in: Biologic Selection in Asthma and COPD

Treatable Traits in Asthma and COPD: Clinical Forum Perspectives

Treatable Traits in Asthma and COPD: Clinical Forum Perspectives

Biologic Access in Asthma and COPD: Clinical Forum Perspectives

Biologic Access in Asthma and COPD: Clinical Forum Perspectives

FDA Grants Fast Track Status to EG-501 in Neuropsychiatric Lupus Marco Meglio

The designation follows phase 2 ClearMEMory data in which the oral NMDA receptor antagonist improved objective cognitive performance over placebo at 12 weeks.

New Phase 1 Study Tests Intrathecal Gene Therapy Agent INS1201 in Duchenne Muscular Dystrophy Marco Meglio

The phase 1 ASCEND trial is testing a single intrathecal dose of the investigational AAV9 gene therapy INS1201 in young boys with Duchenne muscular dystrophy.

Satralizumab Reduces MOGAD Relapse Risk in Phase 3 METEOROID Trial Louie Pasculli

Findings from the phase 3 METEOROID trial showed that satralizumab reduced the risk of relapse in patients with relapsing myelin oligodendrocyte glycoprotein antibody-associated disease.

TSHA-102 Produces Multidomain Functional Gains in Rett Syndrome Matt Hoffman

All 10 REVEAL Part A participants gained or regained at least 1 developmental milestone after a single intrathecal TSHA-102 dose, with no treatment-related SAEs reported.

Todays Updates

Clinical Forum Weighs in: Biologic Selection in Asthma and COPD

Clinical Forum Weighs in: Biologic Selection in Asthma and COPD

Treatable Traits in Asthma and COPD: Clinical Forum Perspectives

Treatable Traits in Asthma and COPD: Clinical Forum Perspectives

Biologic Access in Asthma and COPD: Clinical Forum Perspectives

Biologic Access in Asthma and COPD: Clinical Forum Perspectives

FDA Grants Fast Track Status to EG-501 in Neuropsychiatric Lupus Marco Meglio

The designation follows phase 2 ClearMEMory data in which the oral NMDA receptor antagonist improved objective cognitive performance over placebo at 12 weeks.

New Phase 1 Study Tests Intrathecal Gene Therapy Agent INS1201 in Duchenne Muscular Dystrophy Marco Meglio

The phase 1 ASCEND trial is testing a single intrathecal dose of the investigational AAV9 gene therapy INS1201 in young boys with Duchenne muscular dystrophy.

Satralizumab Reduces MOGAD Relapse Risk in Phase 3 METEOROID Trial Louie Pasculli

Findings from the phase 3 METEOROID trial showed that satralizumab reduced the risk of relapse in patients with relapsing myelin oligodendrocyte glycoprotein antibody-associated disease.

TSHA-102 Produces Multidomain Functional Gains in Rett Syndrome Matt Hoffman

All 10 REVEAL Part A participants gained or regained at least 1 developmental milestone after a single intrathecal TSHA-102 dose, with no treatment-related SAEs reported.

Latest News

The Galassia trial will evaluate the safety and tolerability of intranasal cenegermin in about 60 children with spastic cerebral palsy across Italy.

New analyses found differences in functional outcomes over 2 years among patients aged 8 to 12 years treated with delandistrogene moxeparvovec, while separate findings characterized micro-dystrophin expression in children younger than 3 years.

An analysis of the phase 3 GEMZ trial presented at CNS 2026 showed that fenfluramine produced comparable seizure reductions and safety outcomes in children and adults with CDKL5 deficiency disorder.

A post hoc analysis of the phase 2/3 NPC002 trial presented at CNS 2026 showed that the benefit of arimoclomol plus miglustat in Niemann-Pick disease type C emerged as early as 3 months after treatment initiation.

The professor of neurology and program director of Neuromuscular Medicine at UC Irvine, discusses phase 3 NIMBLE trial findings for cemdisiran in generalized myasthenia gravis, including its novel siRNA mechanism, efficacy, and hospitalization data.

New 48-week phase 3 data show many patients with generalized myasthenia gravis reached minimal symptom expression on telitacicept, and most who did sustained it.

News All Time

Brain Chat: The Overlap Years: Perimenopause, Menopause, and MS Mitzi Joi Williams, MD

Mitzi Joi Williams, MD, is joined by Jakai McEwen, DO, and Charnetta Colton-Poole, MD, to discuss the challenges of communication between patients and their health care teams, including navigating medical language, promoting shared decision-making, and advocating for clear, patient-centered care.

New Study Reveals Plasma Biomarkers Highlight Distinct Biological Responses to Lecanemab in Alzheimer Disease Louie Pasculli, Carlos Cruchaga, PhD

Carlos Cruchaga, PhD, director of the NeuroGenomics and Informatics Center at Washington University, discusses emerging blood-based biomarkers for monitoring biological and cognitive responses to lecanemab treatment in Alzheimer disease.

Christen Kutz, PhD, PA-C, on What Real-World Data Reveal About Treatment Transitions to Nipocalimab in Generalized Myasthenia Gravis Marco Meglio, Christen Kutz, PhD, PA-C

Christen Kutz, PhD, PA-C, discusses a retrospective survey of neurology providers examining why patients with generalized myasthenia gravis are transitioning to nipocalimab and what real-world outcomes data suggest about its role in the treatment landscape.

Advanced Therapies in Parkinson Disease: Matching the Right Treatment to the Right Patient Hubert Fernandez, MD, Louie Pasculli

An MDS 2026 plenary speaker discusses the evolving role of infusion therapies, deep brain stimulation, and focused ultrasound in Parkinson disease, including when to consider device-aided therapies and how treatment selection can be individualized.

From Cord Blood to Cannabinoids: 7 Agents to Watch in Cerebral Palsy Marco Meglio

Seven investigational cerebral palsy therapies—from baby-tooth stem cells to cannabinoids and botulinum toxin—show mixed early results, highlighting key clinical trial milestones ahead.

GLP-1/GIP Agonist KP405 Shows Biomarker Signal in Parkinson Disease Marco Meglio

Exploratory extracellular vesicle biomarkers suggested KP405, a brain-penetrant GLP-1/GIP agonist, engaged insulin signaling and neuroinflammation pathways in a small phase 1 study.

12-Month Gluten-Free Diet Linked to Cognitive, Quality-of-Life Improvements in Parkinson Disease Louie Pasculli

Late-breaking findings from MDS 2026 suggest a gluten-free diet may affect cognitive and quality-of-life measures in Parkinson disease, although no significant changes in motor function were observed.

Emerging Advances in Parkinson Disease Ahead of MDS 2026: Benjamin Walter, MD, MBA Benjamin L. Walter, MD, Louie Pasculli

Benjamin Walter, MD, MBA, discusses how advances in biomarker testing and genetic characterization are informing Parkinson disease care, alongside developments in adaptive deep brain stimulation and efforts to identify disease earlier.

NeuroVoices: Kamalini Galvelis, MS, on Why Diverse Cohorts Matter in Replicating a GBA1 Intronic Risk Variant Isabella Ciccone, MPH, Kamalini Galvelis, MS

The associate vice president of clinical research at the Parkinson's Foundation discussed a large-scale replication of rs3115534-G, a noncoding GBA1 variant tied to Parkinson disease in people of African ancestry.

Oral D1/D2 Agonist Lu AF28996 Increases Good ON-Time in Phase 1b Advanced PD Study, Phase 2 DARE2 Trial Begins Isabella Ciccone, MPH

Investigational oral dopamine agonist prodrug boosts good ON-time, cuts OFF-time and dyskinesia, and reduces levodopa in advanced Parkinson disease, as phase 2 begins.

Clinical Forum Highlights Shifting Paradigm in NF1 Plexiform Neurofibroma Care Marco Meglio

At a NeurologyLive Clinical Forum in Philadelphia, Jessica D. Schulte, MD, PhD, of NYU Grossman School of Medicine, discussed how earlier recognition and systemic therapy options are changing the treatment paradigm for patients with NF1-associated plexiform neurofibromas.

This Week on NeurologyLive® — October 5, 2026 Isabella Ciccone, MPH

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

Ecopipam Shows Early and Sustained Tic Reduction in New Pediatric Tourette Syndrome Analyses Louie Pasculli

New analyses from the D1AMOND clinical program found that 69.8% of participants achieved a clinically meaningful reduction in tic severity within 8 weeks of ecopipam treatment, while an interim 18-month analysis showed sustained improvement without new safety signals.

LRRK2 Inhibitor BIIB122 Fails to Slow Early-Stage Parkinson Disease Marco Meglio

Full results from the phase 2b LUMA showed that investigational BIIB122/DNL151 did not slow confirmed clinical worsening in early-stage Parkinson's disease compared with placebo.

Phase 3 PROPEL Trial to Test Imeroprubart in Generalized Myasthenia Gravis Marco Meglio

PROPEL will test imeroprubart, an investigational FcRn blocker, against placebo in adults with mild to severe generalized myasthenia gravis.

Phase 1 Data Link ARV-102 LRRK2 Degradation to Oculomotor and CSF Biomarker Changes in Parkinson Disease Louie Pasculli

Early phase 1 findings showed that the PROTAC LRRK2 degrader ARV-102 was associated with improvements in saccadic hypometria and changes in CSF markers of endolysosomal function, neuroinflammation, synaptic integrity, and axonal guidance in participants with Parkinson disease.

Recommended News For You

Combination treatment may help cut lifelong ibrutinib for chronic lymphocytic leukemia patients

Aspirin Fails to Boost Survival in Colorectal Cancer Trial

Morning Immunotherapy Does Not Boost Lung Cancer Survival

Progress in Early Cancer Diagnosis; Breast Cancer in Younger Women; Diet and Cancer.

What Caused a Man to Develop Friable Bleeding Nodules on His Arms and Hips?

Revolutionary Cancer Tx Could Transform Autoimmune Disease

A First in Total Larynx Transplant; 'Sniffing' Out Lung Cancer; Gen X Cancer Toll

Recommended Articles For You

Community Survivorship Networks for Long-Term Cancer Wellness 

AI Revolutionizes Immunotherapy: Unveiling Biomarkers with Machine Intelligence 

How to Prevent Tumor Lysis Syndrome in Cancer Patients 

Clinical Pathways in Hematology in Clinical Decision-Making 

Whats more on Hidoc Dr.

Medical Updates
KOL Videos
Surveys
Events

Daily news to keep you up to date

Gain valuable insights with our in-depth KOL Video discussions

Participate and win cash vouchers

Get updates on the latest events happening around the world
Featured News
Featured Articles
Featured Events
Featured KOL Videos

1.

Effects of Recombinant Replacement Therapy Explained in Detailed Regarding Genetic Clotting Disorder.

2.

Pirtobrutinib May Be Beneficial in Follicular Lymphoma with Severe Pretreatment.

3.

Kate Middleton Announces Major Cancer Update

4.

New Nanoparticles Can Destroy Undruggable Cancer Proteins

5.

Future of Lab-Developed Tests Still Taking Shape

1.

How Cancer Cells Evade Immune Destruction and the Fight Back

2.

Menstrual Blood Changes in Iron-Deficiency States: Clinical Insights and Evidence-Based Management

3.

HPV-Related Cervical Cancer: Advances in Screening, Preventiofn & Treatment

4.

The Future of Hematology: Innovations Every Doctor Should Know

5.

Reproductive Aging and Fertility Preservation Timing

1.

International Conference on Innovations in Critical Care for Oncology and Cardiology

2.

International Conference on Oncology, Cardiology and Critical Care Policy

3.

International Conference on Oncology, Cancer Prevention and Public Health

4.

International Conference on Cancer Nursing and Rehabilitation Strategies

5.

International Conference on Best Practices in Oncology, Cardiology and Critical Care

1.

Navigating the Complexities of Ph Negative ALL - Part III

2.

How Nutrition Can Support Patients During Cancer Care

3.

Recent Data Analysis for First-Line Treatment of ALK+ NSCLC: A Final Discussion

4.

Recognizing General Warning Signs of Cancer

5.

An Eagles View - Evidence-based discussion on Iron Deficiency Anemia- Further Talks

Fastest Growing Medical Platform for Doctors

Address :
Hidoc Dr. Inc. | Delaware C Corp | 1309 Coffeen Ave. Suite 1200, Sheridan WY, 82801

Phone :
+1-415-463-3094

Email :
anishagadia@hidoc.co

Quick Links

Drug DatabaseDrug InteractionDiseases Database
KOL VideosKnowledge HubQuizzes
Medical UpdatesEventsAbout Us

© Copyright 2026 Hidoc Dr. Inc.

Terms & Conditions - LLP | Inc. | Privacy Policy - LLP | Inc. | Account Deactivation
Home
Profile
Login
bot