artificial intelligence driven nano enhanced stem cell therapy for neurodegenerative diseases from rational design to clinical translation

Original title: “Artificial intelligence-driven nano-enhanced stem cell therapy for neurodegenerative diseases: from rational design to clinical translation.”

when adverse outcomes become public trials in china

Original title: “When Adverse Outcomes Become Public Trials in China.”

mesenteric unicentric castleman s disease misdiagnosed as gastrointestinal neuroendocrine tumour

Original title: “Mesenteric unicentric Castleman's disease misdiagnosed as gastrointestinal neuroendocrine tumour.”

reply to the ethics of unlicensed therapy use in primary biliary cholangitis a counter view

Original title: “Reply to 'The ethics of unlicensed therapy use in primary biliary cholangitis: a counter view'.”

summary of the inaugural renu hope conference and scientific symposium july 23 25 2025 long island new york

Original title: “Summary of the Inaugural ReNU Hope Conference and Scientific Symposium, July 23-25, 2025, Long Island, New York.”

intraosseous sarcoidosis imitating peri implantitis a case study

Original title: “Intraosseous sarcoidosis imitating peri-implantitis: A case study.”

rationale and design of ultrasound applicability in the assessment of patients with fibrosing interstitial lung disease lord a research protocol of a prospective cohort study

Original title: “Rationale and design of uLtrasOund applicability in the assessment of patients with fibRosing interstitial lung Disease (LORD): a research protocol of a prospective cohort study.”

tracing the path of non industry driven medicine development for rare diseases through regulatory interactions at the european medicines agency

Original title: “Tracing the path of non-industry-driven medicine development for rare diseases through regulatory interactions at the European Medicines Agency.”

functional and structural outcomes in paediatric myelin oligodendrocyte glycoprotein antibody associated disease mogad a prospective study

Original title: “Functional and structural outcomes in paediatric myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD): a prospective study.”

lecithin cholesterol acyltransferase deficiency with the finding of zebra bodies a diagnostic challenge in the context of suspected fabry disease

Original title: “Lecithin-cholesterol acyltransferase deficiency with the finding of "zebra bodies": A diagnostic challenge in the context of suspected Fabry disease.”

case report of clippers involving brainstem cerebellum white matter of cerebral hemisphere spinal cord and corpus callosum

Original title: “A case report of CLIPPERS involving brainstem, cerebellum, white matter of cerebral hemisphere, spinal cord and corpus callosum.”

validity and reliability of the turkish version of the parental needs scale for rare diseases pns rd

Original title: “The Validity and Reliability of the Turkish Version of the Parental Needs Scale for Rare Diseases (PNS-RD).”

medicine development and access for rare diseases can we do better

Original title: “Medicine Development and Access for Rare Diseases: Can We Do Better?”

expert designed fact sheets and ai based analysis of patient symptoms to combat diagnostic delays in inherited metabolic diseases

Original title: “Expert-Designed Fact Sheets and AI-Based Analysis of Patient Symptoms to Combat Diagnostic Delays in Inherited Metabolic Diseases.”

The efficacy data included 4 patients who had discontinued their SOC therapy in a time frame of 4 to 11 weeks after being treated with FLT201. Newly updated data from Spur Therapeutics’ phase 1/2 GALILEO-1 clinical trial (NCT05324943), which is evaluating the adeno-associated virus (AAV) vector-based gene therapy avigbagene parvec (also known as FLT201) for the treatment of Gaucher disease, has shown that patients who received the gene therapy have maintained clinical benefit for up to 2 years post treatment.1,2 The data were presented at the European Society of Gene & Cell Therapy (ESGCT) 32nd Annual Congress, held October 7 to 10, in Seville, Spain. They still have unmet needs, their bone complications are ongoing, so I don't think as clinicians we should underestimate how it's impacting the patients, and we should always look at better therapies and explore better options.” · Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy. However, it was noted that even ALT increases within the normal range were considered ADRs because the trial protocol considered such increases an adverse event of special interest. Two patients had ALT increases above the normal range that were considered related to the gene therapy product, but both cases resolved. In one of these patients, it was stated that the antibodies did not have an effect on efficacy. In the other patient, who remains on SOC, early GCase expression and lyso-Gb1 reduction were lost, which was deemed potentially related to anti-GCase antibodies and a COVID-19 infection that occurred in the month before gene therapy treatment.

Original title: “Patients Treated With Spur Therapeutics’ Gaucher Disease Gene Therapy FLT201 Maintain Clinical Benefit for Up to 2 Years | CGTlive®”

ai succeeds in diagnosing rare diseases

Original title: “AI succeeds in diagnosing rare diseases.”

tsutsugamushi hepatopathy a scoping review

Original title: “Tsutsugamushi Hepatopathy: A Scoping Review.”

ablation of an atrioventricular nodal reentrant tachycardia and a focal left atrial tachycardia in azygos continuation

Original title: “[Ablation of an atrioventricular nodal reentrant tachycardia and a focal left atrial tachycardia in azygos continuation].”

automated computational workflow quiche reveals structural definitions of antitumor responses in triple negative breast cancer

Original title: “The automated computational workflow QUICHE reveals structural definitions of antitumor responses in triple-negative breast cancer.”