Author: anna_acnr

FDA approves ISEMBYLD as muscle-targeted treatment for spinal muscular atrophy

Posted on October 15, 2026 by - Spinal muscular atrophy

The US Food and Drug Administration (FDA) has approved ISEMBYLD (apitegromab-mstn) for adults and children aged two years and older with spinal muscular atrophy (SMA) who are receiving a survival motor neuron 2 (SMN2)-targeted treatment. Developed by Scholar Rock, ISEMBYLD is the first muscle-targeted treatment approved for people with SMA.

SMA causes progressive loss of motor neurons and muscle wasting, leading to declining motor function and reduced independence. ISEMBYLD is designed to address the muscular component of the disease by inhibiting myostatin signalling, potentially helping to improve motor function alongside existing SMN2-targeted therapies.

The approval was supported by results from the Phase 3 SAPPHIRE study, a randomised, double-blind, placebo-controlled trial. At one year, participants receiving the recommended 10 mg/kg dose of ISEMBYLD alongside an SMN2-targeted treatment had a 2.2-point improvement in the Hammersmith Functional Motor Scale-Expanded (HFMSE) compared with those receiving background treatment and placebo. The study’s main efficacy population included 103 participants aged two to 12 years.

A clinically meaningful increase of at least three HFMSE points was observed in 34.2% of patients receiving ISEMBYLD, compared with 13.5% receiving placebo. The reported nominal p-value for the primary analysis was 0.0121.

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The safety database includes more than 500 people treated with apitegromab across clinical studies. Common adverse reactions in SAPPHIRE included upper respiratory tract infections, vomiting, cough, viral infections, headache, gastroenteritis, pharyngitis and hypersensitivity. Fractures occurred in 9% of patients receiving 10 mg/kg, compared with 2% in the placebo group.

This news item has been summarised using AI and checked by humans before publication.

Sources

US Food and Drug Administration

FDA Orphan Drug Designations and Approvals

ISEMBYLD prescribing information

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MODAG wins CPHI Pharma Award for Parkinson’s diagnostics and therapy

Posted on October 13, 2026 by - Awards & Appointments, Parkinson’s Disease

German biotechnology company MODAG GmbH has won the CPHI Pharma Award 2026 in the API Development and Innovation category, recognising its approach to combining diagnostics for Parkinson’s disease with the development of a potential disease-modifying treatment.

Based in Wendelsheim, Germany, MODAG received the award on 6 October at CPHI Milan. The company was recognised for bringing together PD DETECT®, a diagnostic test for pathological alpha-synuclein aggregates, and emrusolmin, an investigational drug targeting the same protein. Other finalists included Lonza, Cambrex and FUJIFILM Biotechnologies.

Dr Torsten Matthias. Photo credit: Valentina Mognoni.

The award highlights a shift from the manufacturing and synthesis technologies recognised in previous years towards an approach integrating diagnosis and treatment. MODAG’s strategy aims to identify the underlying pathology in patients while developing a therapy directed at the same disease-associated protein.

Launched in March 2026, PD DETECT® detects pathological alpha-synuclein aggregates in cerebrospinal fluid. According to MODAG, it is the first CE-marked test kit of its kind. Parkinson’s disease diagnosis has traditionally relied largely on clinical symptoms, making biological markers potentially valuable for research and patient selection in clinical trials.

CPHI Award Winners. Photo Credit: Valentina Mognoni.

Emrusolmin is a small-molecule candidate designed to selectively target pathological alpha-synuclein oligomers. Developed in collaboration with Teva, it is being evaluated in a Phase 2 clinical trial for multiple system atrophy (MSA), a rare and progressive neurodegenerative disorder that shares features with Parkinson’s disease. The US Food and Drug Administration granted the programme Orphan Drug designation in 2022 and Fast Track designation in September 2025.

MODAG chief executive and co-founder Dr Torsten Matthias said that combining diagnostics and treatment could help researchers establish whether patients have the relevant disease pathology before entering clinical trials. The company also acknowledged the contributions of its partners, including Teva and the Michael J. Fox Foundation, which has supported its research since 2014 with more than US$7 million.

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Founded in 2013, MODAG develops diagnostic tools and potential disease-modifying treatments for synucleinopathies, including Parkinson’s disease and MSA, as well as other neurodegenerative conditions.

The award recognises an integrated approach that could support more targeted clinical research, although emrusolmin remains investigational and its clinical effectiveness has yet to be established.

This news item has been summarised using AI and checked by humans before publication.

Sources

MODAG GmbH

CPHI

US Food and Drug Administration

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New trial aims to halt Parkinson’s disease before its onset

Posted on October 12, 2026 by - Parkinson’s Disease

A new clinical trial led by researchers at Yale School of Medicine is investigating whether Parkinson’s disease can be slowed or prevented before motor symptoms appear. The PRISMS study is testing the anti-inflammatory drug adalimumab in people with REM sleep behaviour disorder (RBD), a condition that can precede Parkinson’s disease by many years.

RBD causes people to physically act out their dreams because the normal mechanism that prevents movement during REM sleep is disrupted. In people with isolated RBD, the risk of developing Parkinson’s disease or a related neurological disorder is substantially increased. Researchers estimate that the transition can occur over a period of 15 to 20 years, creating a potential window for early intervention.

The Yale team believes inflammation may play an important role during this early phase of disease. Studies of spinal fluid from people with RBD found increased white blood cell activity and evidence of inflammatory processes involving tumour necrosis factor (TNF). Adalimumab, already used to treat several inflammatory and autoimmune conditions, blocks TNF and could potentially reduce immune-related damage to vulnerable nerve cells.

The PRISMS study is recruiting adults aged 50 to 80 with confirmed RBD and reduced sense of smell, two features that together are associated with a high likelihood of underlying alpha-synuclein pathology. Participants will receive either adalimumab or placebo every two weeks for two years, alongside assessments of movement, cognition, immune activity and Parkinson’s-related biomarkers.

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Researchers will also use brain imaging and, in some participants, spinal fluid analysis to assess whether treatment affects early changes associated with Parkinson’s disease. The study is being conducted across centres in the United States and Canada.

The trial does not yet establish that adalimumab can prevent Parkinson’s disease. However, the researchers hope it will demonstrate whether targeting inflammation before motor symptoms emerge could alter the disease trajectory and encourage further trials of disease-modifying treatments.

This news item has been summarised using AI and checked by humans before publication.

Sources

Yale School of Medicine

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EU regulators recommend approval of infusion therapy for pediatric MS

Posted on October 9, 2026 by - Multiple Sclerosis

EU regulators have recommended approval of Ocrevus (ocrelizumab) for children and teenagers aged 10 and older with relapsing forms of multiple sclerosis (MS), potentially expanding access to a high-efficacy treatment for paediatric-onset MS.

The recommendation was issued by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). The European Commission will now review the recommendation and make the final decision on whether to approve the treatment for this age group. A decision is expected in the near future.

Paediatric-onset MS is relatively uncommon, but young people with the condition can experience frequent and severe relapses. Treatment options have historically lagged behind those available for adults because many MS therapies have not been formally studied in children.

Ocrevus is an antibody therapy that targets B-cells, immune cells involved in the inflammation associated with MS. It is administered by intravenous infusion, with the first two infusions given two weeks apart and subsequent treatments administered every six months.

The proposed European indication is supported by results from the Phase 3 OPERETTA 2 clinical trial, which included nearly 200 children and adolescents with paediatric-onset MS. Participants were randomly assigned to receive Ocrevus or Gilenya (fingolimod), an oral treatment already approved for paediatric MS.

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Over the two-year study, participants treated with Ocrevus experienced fewer relapses than those receiving Gilenya. Safety findings were also consistent with the established safety profile of Ocrevus in adults.

Ocrevus is already approved in the EU for certain adults with relapsing MS and primary progressive MS. In the US, the treatment has also been authorised for children aged 10 and older with relapsing-remitting MS.

If the European Commission follows the CHMP recommendation, Ocrevus would provide another high-efficacy treatment option for young people with relapsing MS in the EU. The CHMP opinion applies to the intravenous formulation, rather than the under-the-skin formulation available for adults in some markets.

This news item has been summarised using AI and checked by humans before publication.

Sources

Multiple Sclerosis News Today

Roche press release

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Viral infections may accelerate ALS progression, study finds

Posted on October 5, 2026 by - Amyotrophic Lateral Sclerosis (ALS)

Common viral infections may accelerate the onset and progression of amyotrophic lateral sclerosis (ALS), according to new research from McMaster University. The findings suggest that the immune response triggered by infections, rather than direct infection of nerve cells, could contribute to faster disease progression.

Researchers studied animal models of ALS following infection with either influenza A virus or SARS-CoV-2, the virus responsible for COVID-19. Compared with uninfected animals, those exposed to either virus experienced a more rapid decline in motor function.

The researchers investigated the mechanisms underlying this association and found that respiratory viral infections caused persistent changes in the nervous system. Although neither influenza A nor SARS-CoV-2 needs to infect neurons to produce these effects, both infections triggered an inflammatory response in immune cells within the nervous system.

This process, known as gliosis, remained elevated in the spinal cord after the infections had been cleared. Gliosis is already recognised as an important feature of ALS, and the researchers suggest that infection-related inflammation could amplify this process and contribute to faster disease progression.

The study also found evidence that interventions targeting the infection or inflammatory response could influence disease progression in the animal models. Treatment with antiviral medicines and suppression of gliosis using anti-inflammatory treatment were both associated with a significant reduction in the rate of ALS progression.

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The researchers said the findings provide a possible mechanistic explanation for previous observations linking viral infections with neurodegenerative disease. They also suggest that preventing or limiting infections could potentially have benefits beyond reducing the immediate effects of infection.

However, the findings are based on pre-clinical animal models and do not establish that viral infections accelerate ALS progression in people. Further research will be needed to determine whether the mechanisms identified in these models apply to human disease and whether antiviral or anti-inflammatory strategies could have therapeutic value.

This news item has been summarised using AI and checked by humans before publication.

Sources

McMaster University via Newswise

Nature Communications

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Stroke rates nearly double among younger adults over three decades

Posted on October 2, 2026 by - Stroke

Stroke rates among adults aged 20 to 54 nearly doubled over a 27-year period in a large US population study, with the increase driven primarily by ischaemic strokes. The findings also show that more younger adults who experience stroke are surviving the acute event, potentially increasing the number living with long-term disability.

Researchers examined first-ever strokes among 2,076 adults aged 20 to 54 in the Greater Cincinnati and Northern Kentucky region between 1993 and 2020. The overall stroke rate increased from 33.9 cases per 100,000 person-years in 1993-1994 to 62.2 in 2020.

The increase was largely attributable to ischaemic stroke, which occurs when blood flow to part of the brain is blocked. Rates of ischaemic stroke rose from 23.8 to 47.3 cases per 100,000 person-years over the study period. No comparable increase was identified for intracerebral haemorrhage or subarachnoid haemorrhage.

Several health conditions and behaviours also became more common among younger adults experiencing stroke. Rates of high blood pressure, diabetes and atrial fibrillation increased over time, alongside substance use. Substance use rose from 4.6% of younger adults with stroke in 1993-1994 to 40.2% in 2020, with much of the increase reflecting marijuana use.

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Despite the rise in stroke incidence, 30-day mortality declined modestly after adjustment for age, race and sex, falling from 11.7% in 1993-1994 to 9.4% in 2020. The improvements were largely observed among people with intracerebral haemorrhage and subarachnoid haemorrhage.

The researchers said the findings highlight the importance of identifying and managing stroke risk factors earlier in adulthood. However, the study could not determine whether changes in risk factors directly explain the increase in stroke rates because comparable information was not available for people without stroke.

This news item has been summarised using AI and checked by humans before publication.

Sources

Neurology

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New metagenomic approach aims to improve detection of meningitis and encephalitis infections

Posted on September 29, 2026 by - Encephalitis

Researchers in Birmingham have developed a new approach that could improve the detection of infections affecting the central nervous system, including meningitis and encephalitis, by combining metagenomic sequencing with computational methods designed to distinguish genuine pathogens from background signals.

The research, led by Dr Ghaniah Hassan-Smith of Aston Medical School and University Hospitals Birmingham, addresses the difficulty of identifying the causative organism in many suspected central nervous system infections. Conventional testing typically involves cerebrospinal fluid obtained through lumbar puncture, followed by microscopy, culture and targeted PCR tests based on the organisms clinicians suspect.

However, in some settings, including intensive care, the causative pathogen is not identified in more than half of patients with suspected central nervous system infection. This can occur when an unexpected organism is responsible, pathogen levels are low, or treatment has already begun.

The researchers explored metagenomic sequencing, which analyses genetic material in a sample rather than testing for individual organisms. This can potentially detect a broad range of bacteria, viruses and other microorganisms, including unexpected causes of infection.

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A key challenge is that sequencing can also detect microbial material originating from contamination, including skin organisms or laboratory reagents. The team therefore developed a series of bioinformatics filters to help distinguish potentially significant pathogen signals from this background noise.

The method was tested using cerebrospinal fluid samples from patients with central nervous system infections treated at University Hospitals Birmingham and compared with samples from people with non-infectious neurological conditions. The approach combines multiple sequencing signals rather than relying on a single finding.

The researchers emphasised that metagenomic sequencing is not intended to replace conventional microbiology. Instead, the work aims to support more standardised interpretation of sequencing results and help bring the technology closer to routine clinical use.

The study, published in BMC Infectious Diseases, was a collaboration involving Aston University, University Hospitals Birmingham and the University of Birmingham.

This news item has been summarised using AI and checked by humans before publication.

Sources

Aston University

BMC Infectious Diseases

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AAN and AHS update guidance on migraine prevention medications for adults

Posted on September 23, 2026 by - Headache

The American Academy of Neurology (AAN) and American Headache Society (AHS) have issued an updated joint practice guideline to help clinicians select preventive medications for adults with migraine. The evidence-based guidance updates recommendations published in 2012 and covers both established treatments and newer preventive therapies.

The guideline recommends offering preventive treatment to people experiencing four or more migraine days per month, or four or more moderate to severe headache days per month. Prevention should also be considered when migraine substantially affects a person’s ability to work, carry out daily activities or otherwise function normally.

Migraine is a chronic neurological disease that can cause headache, nausea, sensitivity to light and sound, dizziness, visual symptoms and cognitive difficulties. Attacks may last from several hours to several days. The guideline addresses both episodic and chronic migraine, with chronic migraine defined by headache on at least 15 days per month, including at least eight days with migraine features.

The update reflects the expansion of preventive treatment options since the previous AAN and AHS guideline. Clinicians are advised to consider the impact of migraine on quality of life and discuss the strength of evidence for individual treatments, potential adverse effects, costs and different routes and frequencies of administration.

Treatment choice should also take account of coexisting conditions. Some established preventive medicines, including certain antidepressants and blood pressure treatments, may address migraine alongside another condition, while newer therapies have been developed specifically for migraine.

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The guideline emphasises shared decision-making and notes that failure to respond adequately to one preventive medication does not mean another approach will be ineffective. Clinicians should assess treatment effectiveness after initiation according to the recommended timeframes.

The joint guideline is due to be published in Neurology and Headache and has been endorsed by the American Academy of Family Physicians. It was funded by the AAN.

This news item has been summarised using AI and checked by humans before publication.

Sources

American Academy of Neurology / Newswise

American Headache Society clinical practice guidelines

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Early MS treatments differ most in relapses and MRI lesions, not disability

Posted on September 18, 2026 by - Multiple Sclerosis

Early treatment choices for people newly diagnosed with multiple sclerosis (MS) may influence relapse rates and MRI findings, but a two-year study found no clear differences in disability outcomes between treatment groups.

The study, published in Neurology Open Access, followed 509 people with newly diagnosed relapsing or progressive MS who had not previously received disease-modifying therapy. Participants had an average age of 33 and were followed for two years.

Participants were grouped according to their first treatment. These included injectable and oral platform therapies, high-efficacy treatments, B-cell-depleting therapies, or no treatment.

The strongest differences were seen among people receiving B-cell-depleting therapies, including rituximab and ocrelizumab. This group had an average relapse rate of 0.04 per year, compared with 0.16 among those receiving oral platform therapies. After adjustment for factors including age, sex and disease duration, B-cell treatment was associated with a 62% lower relapse rate.

B-cell-depleting treatment was also associated with a small reduction in MRI lesion volume compared with oral platform therapies. Other high-efficacy treatments showed a similar trend, although the difference was not statistically significant.

Treatment persistence also differed. After two years, 94% of people receiving B-cell therapies remained on their initial treatment, compared with 87% receiving high-efficacy therapies, 72% taking oral platform treatments and 61% taking injectable platform therapies.

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However, researchers found no differences between treatment groups in disability score changes or blood levels of serum neurofilament light chain, a marker of nerve cell injury. The findings therefore suggest that early treatment differences may be more apparent in inflammatory disease activity than in disability over the first two years.

The researchers emphasised that treatment decisions should be individualised. Because participants were not randomly assigned to treatments, the study cannot establish that one treatment causes better outcomes. Longer follow-up will be needed to determine whether differences in relapse and MRI activity translate into differences in disability over time.

This news item has been summarised using AI and checked by humans before publication.

Sources

American Academy of Neurology via Newswise

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FDA clears Roche blood test to assess Alzheimer’s disease amyloid pathology

Posted on September 16, 2026 by - Alzheimer's Disease

The US Food and Drug Administration (FDA) has cleared Roche’s Elecsys pTau217 blood test to help assess the likelihood of amyloid pathology in adults aged 55 and over being evaluated for Alzheimer’s disease.

The test measures plasma phosphorylated tau 217 (p-tau217), a biomarker strongly associated with amyloid pathology. Roche describes Elecsys pTau217 as the first FDA-cleared blood test using a single biomarker to support both rule-in and rule-out assessment of amyloid pathology across primary and specialist care.

The test is not a stand-alone diagnostic for Alzheimer’s disease. Results are intended to be considered alongside a patient’s clinical history, cognitive assessment and other diagnostic information. It produces three possible classifications: positive, indicating a high likelihood of amyloid pathology; negative, indicating a low likelihood; and intermediate, for which further confirmatory testing is recommended.

The assay’s two cut-offs were validated against amyloid PET in early-stage disease cohorts in both primary and specialist care settings. However, its safety and effectiveness have not been established for predicting future dementia, use in other neurological conditions or monitoring treatment response.

The clearance comes as blood-based biomarkers are increasingly being investigated as more accessible alternatives to established methods such as amyloid PET and cerebrospinal fluid testing, which can be costly, invasive or less readily available outside specialist centres.

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Elecsys pTau217 runs on Roche’s cobas immunoassay systems, with an analytical run time of approximately 18 minutes. Labcorp and Quest Diagnostics have announced plans to offer the test, with Quest expecting to introduce a laboratory service based on the assay during the fourth quarter of 2026.

The test differs from another FDA-cleared assay, Fujirebio’s Lumipulse G pTau 217/β-amyloid 1-42 Plasma Ratio, which uses two biomarkers to calculate a ratio. The new clearance adds to Roche’s expanding portfolio of blood and cerebrospinal fluid biomarkers for Alzheimer’s disease.

This news item has been summarised using AI and checked by humans before publication.

Sources

Roche Diagnostics

NeurologyLive

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