RUSH UNIVERSITY MEDICAL CENTER
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- -
- Established
- 1837-01-01
- Employees
- -
- Market Cap
- -
- Website
- https://www.rush.edu/
QurAlis Secures Exclusive License for Novel RNA-Targeted Therapy in Fragile X Syndrome
• QurAlis has licensed a groundbreaking RNA-targeted mechanism from UMass Chan Medical School that restores functional FMRP protein in Fragile X syndrome patients.
• The company's preliminary data suggests a biomarker can detect FMR1 mis-splicing, potentially enabling precision medicine approaches for up to 80% of FXS patients.
• QurAlis is applying its FlexASO® platform to advance FMR1-217 as a therapeutic target, with plans to nominate a candidate for IND-enabling studies in the near future.
CareDx Expands AlloSure Testing to Pediatric Heart and Simultaneous Pancreas-Kidney Transplant Patients
• CareDx has received New York State Clinical Laboratory Evaluation Program approval for AlloSure testing in pediatric heart transplant patients of all ages, including infants, and in simultaneous pancreas-kidney transplant recipients.
• Studies show AlloSure Heart can detect rejection in pediatric patients with performance consistent with adults, demonstrating an 81% reduction in surveillance biopsies in a prospective study.
• The expanded indications address critical needs in transplant care, particularly for the approximately 450 pediatric heart transplants and 700 simultaneous pancreas-kidney transplants performed annually in the U.S.
Leukocyte Count Emerges as Predictor for Long COVID Severity in Postmenopausal Women
• New research published in Menopause reveals that higher leukocyte counts are strongly associated with increased severity of long COVID symptoms in older postmenopausal women.
• The study analyzed data from over 1,200 Women's Health Initiative participants, finding that white blood cell count serves as an independent predictor of post-acute sequelae of SARS-CoV-2 infection (PASC) severity.
• This discovery could transform diagnosis and treatment approaches for long COVID, offering healthcare providers a widely available, inexpensive clinical marker to identify at-risk patients.
NCL Translational Research Conference Highlights Advances in Batten Disease Therapies
• The NCL Translational Research Conference addressed challenges in Batten disease clinical trials and emphasized stakeholder collaboration.
• Discussions focused on leveraging therapeutic platforms, real-world data, and AI to improve Batten disease diagnosis and patient care.
• Advanced therapeutic strategies, including gene therapies and personalized antisense oligonucleotides, were explored for various Batten disease subtypes.
• The conference underscored the importance of natural history studies and biomarker development for advancing trial readiness in Batten disease.
Gene Therapies Show Promise in Targeting the Root Cause of Rett Syndrome
• Two companies, Neurogene and Taysha Gene Therapies, are in Phase 1/2 clinical trials with gene therapies targeting the MECP2 gene for Rett syndrome.
• These therapies aim to address the underlying genetic cause of Rett syndrome, unlike existing treatments that only manage symptoms.
• Both companies have incorporated regulatory mechanisms to ensure precise MECP2 expression, avoiding complications from overexpression.
• Preliminary data from ongoing trials are expected in late 2024 and early 2025, offering hope for a potential disease-modifying treatment.
PSMD Neuroimaging Marker Shows Promise in Identifying Dementia Risk
• A new neuroimaging marker, peak-width of skeletonized mean diffusivity (PSMD), correlates with general cognition and may identify individuals at risk of dementia.
• The study found that higher PSMD values were associated with lower general cognitive function, independent of age, sex, education and intracranial volume.
• PSMD demonstrates excellent instrumental properties and biological validation, making it a potential biomarker for clinical trials related to vascular contributions to cognitive impairment and dementia (VCID).
• Researchers suggest PSMD's non-invasive, automated, and reliable nature makes it ideal for assessing cerebral small vessel diseases in dementia studies.
ReNu Shows Promise in Phase 3 Trial for Knee Osteoarthritis
• Organogenesis Holdings reports positive interim analysis from its Phase 3 trial of ReNu for knee osteoarthritis, covering 50% of 474 planned patients.
• The Data Monitoring Committee recommended the trial proceed without modifications, confirming favorable results and a consistent safety profile for ReNu.
• The fully enrolled trial with 594 patients evaluates ReNu versus saline control via intra-articular injection, measuring knee pain reduction using the WOMAC pain scale.
• Knee osteoarthritis affects approximately 31.1 million Americans, with projections estimating a rise to 34.4 million cases by 2027.
Cretostimogene Shows Durable Responses in High-Risk Bladder Cancer
• Cretostimogene monotherapy demonstrated a 74.5% complete response rate at any time in patients with high-risk BCG-unresponsive NMIBC.
• The median duration of response has not been reached but exceeds 27 months, indicating a sustained treatment effect.
• The BOND-003 trial showed a favorable safety profile with no Grade 3 or higher treatment-related adverse events reported.
• 97.3% of patients were free from progression to muscle-invasive bladder cancer at 12 months, highlighting the therapy's potential to prevent disease advancement.
EUS-Guided Celiac Plexus Block Shows Promise in Pancreatic Cancer Pain Management
• Endoscopic ultrasound-guided celiac plexus block (EUS-CPB) is being investigated for pain palliation in pancreatic cancer patients.
• A multi-center, randomized controlled trial is underway to evaluate the efficacy of EUS-CPB compared to standard pain management.
• The study aims to determine if EUS-CPB can reduce pain intensity and improve quality of life for patients with pancreatic cancer.
• Preliminary results suggest EUS-CPB could offer a valuable alternative or adjunct to traditional pain control methods.
Hepatology Review: Advancements in MASH, PBC Treatments and Disease Risk
• ALG-055009, a thyroid hormone receptor beta agonist, demonstrated statistically significant reductions in liver fat in a Phase 2a study for MASH, showing promise for MASH resolution and fibrosis improvement.
• New Phase 2a data suggests CNP-104 could be a novel treatment for PBC by targeting the root cause of the disease, inducing tolerance to pathogenic T-cells and improving liver health.
• Research indicates that MASLD and MetALD are associated with an increased risk of liver and gastrointestinal cancers, emphasizing the need for vigilance and preventive measures.
• The European Commission revoked the marketing authorization for obeticholic acid (Ocaliva) in Europe for PBC treatment following concerns about its benefit-risk profile.
Zatolmilast Shows Promise in Treating Fragile X Syndrome
• Zatolmilast, an experimental drug, has shown potential in improving cognitive and social functions in individuals with Fragile X syndrome.
• A 2021 study indicated that zatolmilast improved memory and language skills in adult males with Fragile X over a 12-week period.
• Two ongoing larger studies are evaluating zatolmilast's efficacy, potentially leading to the first FDA-approved treatment for Fragile X syndrome.
• The drug targets an enzyme involved in memory and cognitive impairment, offering a new approach to managing Fragile X symptoms.
FDA Approves First Treatments for Niemann-Pick Type C, Offering New Hope to Patients
• The FDA has approved two new treatments for Niemann-Pick Type C, a rare and fatal genetic disorder, marking a significant milestone for patients and families.
• Miplyffa (arimoclomol) by Zevra Therapeutics, in combination with miglustat, is approved for neurological symptoms in patients aged 2 and older.
• Aqneursa (levacetylleucine) by IntraBio Inc. has also been approved as a stand-alone therapy for neurological manifestations of Niemann-Pick disease type C.
• These approvals underscore the FDA's commitment to supporting the development of new treatments for rare diseases, providing hope for improved management of symptoms.
Cretostimogene Shows High Complete Response Rate in BCG-Unresponsive Bladder Cancer
• Cretostimogene monotherapy achieved a 75.2% complete response rate in patients with high-risk, BCG-unresponsive non-muscle invasive bladder cancer (NMIBC).
• Durable responses were observed, with 29 patients maintaining complete response for 12 months or longer, suggesting a sustained therapeutic effect.
• The Phase 3 BOND-003 trial reported no Grade 3 or higher treatment-related adverse events, highlighting a favorable safety profile.
• Cretostimogene has received Fast Track and Breakthrough Therapy designations from the FDA, potentially expediting its path to regulatory approval.
FDA Grants Priority Review to IntraBio's IB1001 for Niemann-Pick Disease Type C
• The FDA has accepted IntraBio's New Drug Application for IB1001, granting Priority Review for Niemann-Pick Disease Type C treatment.
• IB1001's NDA is supported by positive Phase 3 trial results, demonstrating improvements in neurological symptoms and quality of life.
• A PDUFA target action date is set for September 24, 2024, potentially expediting the availability of this oral therapy.
• IntraBio secured over $40 million in equity financing to support the commercialization of IB1001, pending FDA approval.
MIND Diet Shows Limited Short-Term Cognitive Benefits in Clinical Trial
• A clinical trial evaluating the MIND diet, designed to boost brain health, showed improvements in brain structure but similar gains in the control group.
• The three-year study found that both the MIND diet group and the control group experienced improvements in cognitive function and weight loss.
• Experts suggest the trial duration may have been too short to observe significant differences, and that increased awareness of healthy eating impacted both groups.
• The MIND diet incorporates elements of the Mediterranean and DASH diets, emphasizing foods known to support brain health.
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