Latest news with #PeripheralNerveSociety
Business Insider
18-05-2025
- Health
- Business Insider
Intellia Therapeutics announces two-year follow-up data from trial of nex-z
Intellia Therapeutics (NTLA) announced positive two-year follow-up data from the ongoing Phase 1 trial of investigational nexiguran ziclumeran, nex-z, for the treatment of hereditary ATTR amyloidosis with polyneuropathy. Results were shared in an oral presentation on Sunday, May 18 at the 2025 Peripheral Nerve Society, PNS, Annual Meeting in Edinburgh, United Kingdom. The Phase 3 MAGNITUDE-2 trial design of nex-z in ATTRv-PN was also exhibited in a poster presentation. Across patients who received a one-time dose of 0.3 mg/kg or higher, the mean serum TTR reduction by Day 28 was 90%, with levels remaining virtually unchanged for at least 24 months. Favorable trends indicating stability or improvement were observed in patients with ATTRv-PN, including six patients previously on patisiran for a mean of 5.5 years, who had evidence of disease progression prior to entering the study. Stability or improvement was based on evaluation of multiple clinical and biomarker measures, including Neuropathy Impairment Score, modified Neuropathy Impairment Score +7, modified BMI, Norfolk Quality of Life-Diabetic Neuropathy questionnaire and neurofilament light chain
Business Insider
18-05-2025
- Business
- Business Insider
Applied announces INSPIRE Phase 2/3 trial primary endpoint not significant
Applied Therapeutics (APLT) presented full 12-month clinical results and new topline data from 18 and 24 months from the INSPIRE Phase 2/3 trial of govorestat for the treatment of Sorbitol Dehydrogenase Deficiency, a subtype of Charcot-Marie-Tooth disease, in an oral presentation at the Peripheral Nerve Society 2025 Annual Meeting, being held May 17-20, 2025 in Edinburgh, Scotland. A reverified, interim analysis of 49 patients evaluable for efficacy as of February 2024, conducted at 12 months of active treatment. Full clinical results from 12 months include: Statistically significant correlation between absolute reduction in sorbitol and change in the 10MWRT and the CMT-FOM Lower Limb domain were observed. The primary endpoint, the 10MWRT, was not statistically significant. Since initiation of the INSPIRE study, the 10MWRT was removed from the CMT-FOM, which now only consists of 11 components. Govorestat treatment demonstrated a statistically significant improvement on key secondary endpoint, CMT-HI at 12 months. A statistically significant correlation between percent change in sorbitol and change in CMT-HI was observed at 12 months for the total score as well as in the mobility and balance domains. Statistically significant lowering of blood sorbitol levels were observed. Govorestat continued to be generally safe and well tolerated, with similar incidence of adverse events between active and placebo-treated groups Confident Investing Starts Here:
Medscape
14-05-2025
- Health
- Medscape
Guillain-Barré Syndrome: When to Suspect, What to Rule Out
The Task Force of the European Academy of Neurology and the Peripheral Nerve Society published updated guidelines in 2024 on the diagnosis and treatment of Guillain-Barré syndrome (GBS), which accounts for approximately 100,000 cases annually. This topic was discussed at the French Language Neurology Days Congress, held in Clermont-Ferrand, France, from 15 to 18 April 2025. The report highlighted that certain infections, particularly those caused by Campylobacter jejuni, could trigger GBS. Furthermore, 'several studies suggest an increased risk following treatments that is associated with immunity, including certain biotherapies such as checkpoint inhibitors or tacrolimus and certain vaccines, such as those for influenza, shingles, or adenoviral vector vaccines against SARS-CoV-2,' summarised Armelle Magot, MD, neurologist at Nantes University Hospital, Nantes, France. The Task Force concluded that the benefits of these vaccines outweigh the rare risk of developing GBS. Diagnostic Criteria The Task Force reviewed various diagnostic criteria proposed in the literature and recommended using the criteria defined in a review published in 2019 as a diagnostic foundation. Key indicators include progressive limb weakness, areflexia or hyporeflexia in the affected areas, and worsening symptoms over more than 4 weeks. Clinical criteria, such as a recent history of infection, cranial nerve involvement, signs of autonomic dysfunction, respiratory failure, or radicular or muscular pain, can further strengthen the diagnostic suspicion. Conversely, certain factors may suggest a differential diagnosis: Persistent asymmetrical weakness, absence of early progression in the initial hours of GBS, predominant sensory signs, fever, and initial disturbances of consciousness. Biologically, serum levels of anti-ganglioside antibodies are generally not necessary, as their diagnostic sensitivity is limited, except in certain conditions, such as Miller Fisher syndrome. Analysis of the cerebrospinal fluid is recommended to rule out uncertain or differential diagnoses. Protein levels without marked hypercellularity are common in GBS, but they are not reliable, and a normal concentration does not exclude the diagnosis, particularly in the first week. However, the presence of a significant white blood cell count (< 50/µL) in the cerebrospinal fluid suggested an alternative diagnosis. Finally, electromyography is a valuable tool for diagnosis, while MRI and ultrasound are more commonly employed in atypical cases to rule out certain differential diagnoses. Management Protocol The Task Force reaffirmed the role of intravenous immunoglobulins (0.4 g/kg/d for 5 days) or plasmapheresis (4-5 sessions over 1-2 weeks) as first-line treatments. The Task Force did not favour one treatment over the other, except for intravenous immunoglobulins in children, recognising that the combination of both has no added benefit. The therapeutic decision is based on the severity of the condition: Treatment should be administered within the first 4 weeks if the patient is unable to walk 10 metres without assistance (GBS disability score ≥ 3). If the patient can walk but cannot run, these treatments may be considered between 2 weeks and 4 weeks if associated severity criteria are present with rapid adverse progression, swallowing difficulties, and so on. Treatment is not recommended for patients with less severe symptoms or those who are asymptomatic without signs of severity. Fluctuations related to treatment may occur in 5%-15% of patients, corresponding to relapses during the plateau phase or after initiating the recovery phase. In such cases, retreatment may be proposed following the same modalities. 'Unfortunately, if no improvement is observed after a week, no second-line treatment can be proposed,' said Aude-Marie Grapperon, MD, neurologist at Marseille University Hospital, Marseille, France. In a clinical trial, the second course of intravenous immunoglobulins showed poor benefit, and, conversely, thromboembolic adverse effects were observed. Corticosteroids are also ineffective, as numerous clinical trials have confirmed their lack of efficacy when used alone or in combination. Innovative Treatments Innovative treatments are currently being evaluated and could provide alternatives for severe forms of the disease. The scientific rationale for these treatments primarily revolves around the early activation of the complement pathway, which is significant in GBS: Eculizumab, a monoclonal anticomplement antibody, has been studied in this context with two encouraging phase 2 studies; however, phase 3 published data indicated no benefits. C1q inhibitors: A phase 1 study demonstrated a favourable safety profile, and the results of a phase 3 study conducted in Asian countries have not yet been published but are reported to be positive. Efgartigimod vs intravenous immunoglobulins is currently in a Phase 2 trial in the United States, which began in September 2024. Imlifidase, an enzyme derived from Streptococcus pyogenes that cleaves immunoglobulin G, functions as a form of chemical plasmapheresis. Open-label studies have suggested a favourable safety profile; however, these findings require validation and publication. Finally, it is essential not to overlook all supportive treatments, such as the treatment of pulmonary infections, psychological support, pain management, and other interventions, which are crucial for the overall improvement of patients. This story was translated from Univadis France using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.
Business Insider
10-05-2025
- Business
- Business Insider
Applied Therapeutics to present full 12-month INSPIRE Phase 3 results
Applied Therapeutics (APLT) announced its late-breaking abstract for govorestat, or AT-007, has been selected for oral presentation at the Peripheral Nerve Society 2025 Annual Meeting, held May 17-20. The presentation will include full 12-month clinical results from the INSPIRE Phase 3 trial in SORD Deficiency, a subtype of Charcot-Marie-Tooth disease. It will also include new topline 18-month and 24-month data. The company previously shared topline 12-month data in February 2024. Protect Your Portfolio Against Market Uncertainty
