
Pivotal AFFINITY DUCHENNE results showed microdystrophin expression above the prespecified threshold in 93% of evaluable participants, with preliminary functional findings favoring treatment over external controls.

Pivotal AFFINITY DUCHENNE results showed microdystrophin expression above the prespecified threshold in 93% of evaluable participants, with preliminary functional findings favoring treatment over external controls.

Three-year extension data from the phase 3 IB1001-301 trial show levacetylleucine slowed disease progression in both adults and children with Niemann-Pick disease type C.

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.

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.

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.

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.

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.

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

The senior vice president of medical and scientific relations at the Alzheimer's Association discussed key themes in Alzheimer disease research presented at AAIC 2026. [WATCH TIME: 8 minutes]

The professor of neurology at the University of Michigan Medical School discussed the themes that defined SLEEP 2026, and what they signal for clinical practice heading into 2027. [WATCH TIME: 5 minutes]

Phase 3 ARISE trial results show solengepras 150 mg significantly reduced OFF time and improved motor and nonmotor symptoms in Parkinson disease.

New cerebrospinal fluid biomarker data suggest shared LRRK2 pathway activation across sporadic and LRRK2-associated Parkinson disease, potentially informing biomarker development and therapeutic targeting.

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.

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.

Phase 2a trial results showed 800 mg vutiglabridin significantly improved motor symptom scores compared with placebo in early-stage Parkinson's disease.

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.

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.

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.

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.

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.

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.

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.

The associate vice president of clinical research at the Parkinson's Foundation discussed new PD GENEration data validating a GBA1 intronic risk variant for Parkinson disease. [WATCH TIME: 6 minutes]

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.

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

New data from a Cure SMA survey showed that children treated with disease-modifying therapy soon after birth walk more often and have fewer comorbidities than those treated later.

A new Bayesian network meta-analysis found nipocalimab's serious adverse event rates compared favorably with other gMG therapies, while overall adverse event rates were comparable.