RSRT

Rett Syndrome Research Trust Partners with Vivalink to Shift Symptom Assessment Paradigms in Rett Syndrome

Retrieved on: 
Dienstag, April 23, 2024
RSRT, Neurological disorder, Health, Rett syndrome, Respiratory rate, Partnership, Research, ECG, Heart rate, Speech, Therapy, Patient, Data collection, Caregiver, Posture, Drug development, Biomarker, Genetic disorder, Sleep, Frasier, Dysautonomia, Medical device

CAMPBELL, Calif., April 23, 2024 /PRNewswire/ -- Vivalink, a leading provider of digital healthcare solutions, announces its collaboration with the Rett Syndrome Research Trust (RSRT) to advance research into Rett syndrome, a rare neurological disorder. As part of this partnership, Vivalink is providing its Multi-Vital ECG wearable technology and data services for heart rate and oxygen saturation monitoring to support RSRT's new VIBRANT study, aimed at assessing autonomic dysfunction in individuals with Rett syndrome.

Key Points: 
  • CAMPBELL, Calif., April 23, 2024 /PRNewswire/ -- Vivalink , a leading provider of digital healthcare solutions, announces its collaboration with the Rett Syndrome Research Trust (RSRT) to advance research into Rett syndrome, a rare neurological disorder.
  • Vivalink's advanced wearable devices provide an important opportunity to expedite drug development in the pursuit of a cure for Rett Syndrome.
  • "We are excited to partner with the Rett Syndrome Research Trust in their pioneering study," said Aaron Timm, Chief Commercial Officer at Vivalink.
  • "We hope to empower researchers with the tools they need to make meaningful advancements in understanding and treating Rett syndrome."

Rett Syndrome Research Trust Launches the MECP2 Editing Consortium With Goal to Advance Programs to Clinical Trials

Retrieved on: 
Mittwoch, April 10, 2024
FDA, Massachusetts Institute of Technology, RSRT, Harvard University, University, Rett syndrome, Gene editing, RNA, MD, Doctor of Philosophy, Research, DNA, Gene, Clinical trial, Pharmaceutical industry

TRUMBULL, Conn. , April 10, 2024 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) has taken a significant stride in the pursuit of innovative genetic medicines for Rett syndrome by establishing the MECP2 Editing Consortium. Mutations in MECP2 cause Rett syndrome, a debilitating neurological disorder that causes a lifetime of severe disabilities. The consortium comprises six distinguished labs, including Adrian Bird, PhD and Jacky Guy, PhD from the University of Edinburgh, Erik Sontheimer, PhD, Jonathan Watts, PhD and Scot Wolfe, PhD from University of Massachusetts Chan Medical School, Guoping Feng, PhD and Chenjie Shen, PhD from Massachusetts Institute of Technology, and Peter Beal, PhD from the University of California, Davis. The editing consortium, supported with a $7 million investment from RSRT, is inspired by the prior success of RSRT's Gene Therapy Consortium which laid the foundation for the current gene therapy trials. RSRT is the largest funder of Rett research with more than $77 million awarded since 2008 to advancing cures.

Key Points: 
  • The Rett Syndrome Research Trust (RSRT) has taken a significant stride in the pursuit of innovative genetic medicines for Rett syndrome by establishing the MECP2 Editing Consortium.
  • TRUMBULL, Conn. , April 10, 2024 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) has taken a significant stride in the pursuit of innovative genetic medicines for Rett syndrome by establishing the MECP2 Editing Consortium.
  • The consortium's inaugural meeting earlier this year set an ambitious agenda to advance editing programs to clinical trials.
  • I'm very much looking forward to working with everyone in the consortium as the programs advance toward the clinic."

ProQR Announces Year End 2023 Operating and Financial Results

Retrieved on: 
Mittwoch, März 13, 2024
Trident, Eli Lilly, Exercise, ProQR, Patient, IP, Therapy, Lilly, RNA editing, Cholestasis, Rett syndrome, Research, Cardiovascular disease, Partnership, Pseudouridine, NTCP, RNA, Functional, Uridine, MECP2, Oligonucleotide, ACTB, LPL, Annual report, Conference, Patent, Biomarker, Mouse, RSRT, Eli Lilly and Company, Liver, ANGPTL3

(Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2023, and provided a business update.

Key Points: 
  • (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2023, and provided a business update.
  • At December 31, 2023, ProQR held cash and cash equivalents of €118.9 million, compared to €94.8 million at December 31, 2022.
  • The Company experienced a net positive cash flow from operating activities in 2023 primarily due to the receipt of the Lilly up-front payment of $60 million in February 2023.
  • For further financial information for the period ended December 31, 2023, please refer to our 2023 Annual Report on Form 20-F and our Statutory Annual Report which will be available on our website, www.

PMD Solutions Supports RSRT's Biosensor VIBRANT Study for Rett Syndrome with Donation of FDA-cleared RespiraSense Devices and Analytics

Retrieved on: 
Donnerstag, März 28, 2024
Emerald, Emory University, Doctor of Philosophy, ECG, Partnership, Respiratory rate, Heart rate, Patient, Research, Neurodevelopmental disorder, RSRT, Dysautonomia, Sleep, Artificial intelligence, Rett syndrome, Tidal volume, Radio, PMD, FDA, Movement, Pharmaceutical industry

TRUMBULL, Conn., March 28, 2024 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is proud to announce its partnership with PMD Solutions, a leading provider of innovative healthcare technologies, in their upcoming biosensor validation study called VIBRANT. PMD Solutions will generously donate their state-of-the-art RespiraSense devices and advanced analytics to support RSRT's efforts in assessing autonomic dysfunction in individuals with Rett syndrome, utilizing cutting-edge digital technologies.

Key Points: 
  • PMD Solutions will generously donate their state-of-the-art RespiraSense devices and advanced analytics to support RSRT's efforts in assessing autonomic dysfunction in individuals with Rett syndrome, utilizing cutting-edge digital technologies.
  • PMD Solutions will generously donate their state-of-the-art RespiraSense devices and advanced analytics to support RSRT's efforts in assessing autonomic dysfunction in individuals with Rett syndrome, utilizing cutting-edge digital technologies.
  • The integration of RespiraSense devices with PMD Solutions' cutting-edge analytics offers a comprehensive solution for tracking and analyzing breathing parameters in Rett syndrome patients.
  • By donating RespiraSense devices and analytics for RSRT's biosensor validation study, PMD Solutions will contribute significantly to the advancement of RSRT's mission to cure Rett syndrome.

The Rett Syndrome Research Trust Strengthens Research Team with Addition of Robert Deans as Chief Technology Officer

Retrieved on: 
Dienstag, Januar 16, 2024
Rubius, Therapy, MD, Knowledge, CRISPR, Rett syndrome, Cell biology, University, Science, Rett, Medicine, Parkinson's disease, IND, Regenerative medicine, Drug development, Doctor of Philosophy, RSRT, Drug, International Society, Microbiology, Pharmaceutical industry

TRUMBULL, Conn., Jan. 16, 2024 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is excited to announce the addition of Robert Deans, PhD, as Chief Technology Officer, Head of Research. Deans brings a wealth of genetic medicine knowledge and hands-on industry experience to RSRT. Most recently, he served as Chief Technology Officer of BlueRock Therapeutics, where he led the charge on stem cell and gene therapy approaches. Deans previously served as Chief Scientific Officer of Rubius Therapeutics and Executive Vice President at Athersys, where he advanced a number of stem cell therapeutics into late-stage clinical development.

Key Points: 
  • The Rett Syndrome Research Trust (RSRT) is excited to announce the addition of Robert Deans, PhD, as Chief Technology Officer, Head of Research.
  • Most recently, he served as Chief Technology Officer of BlueRock Therapeutics, where he led the charge on stem cell and gene therapy approaches.
  • TRUMBULL, Conn., Jan. 16, 2024 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is excited to announce the addition of Robert Deans, PhD, as Chief Technology Officer, Head of Research.
  • Deans' colleagues include Randall Carpenter, MD, Chief Medical Officer; Jana von Hehn, PhD, Chief Scientific Officer, Head of Clinical Development; and Monica Coenraads, Chief Executive Officer.

ProQR and Rett Syndrome Research Trust Join Forces with Axiomer™ RNA Editing Collaboration

Retrieved on: 
Freitag, Januar 5, 2024
RNA, Methyl group, Partnership, Rett syndrome, Research, ProQR, UC, Eon, RSRT, CNS, University of California, Davis, MECP2, Pharmaceutical industry

“The Rett Syndrome Research Trust is the leading patient advocacy group championing a cure for Rett syndrome and we look forward to collaborating with them to further expand the broad applicability of our Axiomer RNA editing technology to Rett syndrome, a rare neurodevelopment disorder with significant unmet medical need,” said Daniel A. de Boer, Chief Executive Officer of ProQR.

Key Points: 
  • “The Rett Syndrome Research Trust is the leading patient advocacy group championing a cure for Rett syndrome and we look forward to collaborating with them to further expand the broad applicability of our Axiomer RNA editing technology to Rett syndrome, a rare neurodevelopment disorder with significant unmet medical need,” said Daniel A. de Boer, Chief Executive Officer of ProQR.
  • “Axiomer has the potential to restore precise levels of the MECP2 protein, which is lacking in Rett syndrome.
  • It is the intent of the partnership to be continued by an expanded co-funding arrangement following the initial discovery work.
  • The co-funding of the next phase of the collaboration would enable clinical development of an Axiomer-based therapeutic for Rett syndrome MECP2.

Rett Syndrome Research Trust Launches the Rett Syndrome Global Registry: A Pioneering Parent-Reported Platform Designed to Expedite the Development of Genetic Medicines

Retrieved on: 
Freitag, November 10, 2023
Consensus, Neurogenic claudication, Human, Rett, Registry, Consensus decision-making, Rett syndrome, Policy, CARE, Therapy, Cystic fibrosis, Baylor University, Clinical trial, Database, FDA, Baylor College of Medicine, RSRT, SHARE, FARA, Parent, WEAR, Inclusive, Vanderbilt University, Roger Hawkins (drummer), FAST, Code, Doctor of Philosophy, Patient, Quality of life, Knowledge, Cystic Fibrosis Foundation, Drug development, Pharmaceutical industry, Medical device, Research, Matrix

TRUMBULL, Conn., Nov. 9, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is excited to announce the launch of the Rett Syndrome Global Registry, a fully-remote platform for parents to share their knowledge and experiences caring for loved ones with Rett syndrome. Inclusivity is a vital component of the Rett Global Registry. It is accessible to families regardless of geographic location, access to medical facilities, or socioeconomic status. Designed to advance the development of genetic medicines, the Rett Global Registry adheres to FDA guidance documents and complies with regulatory requirements. Biopharmaceutical companies pursuing therapeutic programs for Rett are urgently waiting to analyze data collected from the Rett Global Registry. Parents of children and adults with Rett syndrome are encouraged to register as soon as possible.

Key Points: 
  • Designed to advance the development of genetic medicines, the Rett Global Registry adheres to FDA guidance documents and complies with regulatory requirements.
  • Biopharmaceutical companies pursuing therapeutic programs for Rett are urgently waiting to analyze data collected from the Rett Global Registry.
  • TRUMBULL, Conn., Nov. 9, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is excited to announce the launch of the Rett Syndrome Global Registry, a fully-remote platform for parents to share their knowledge and experiences caring for loved ones with Rett syndrome.
  • Designed to advance the development of genetic medicines, the Rett Global Registry adheres to FDA guidance documents and complies with regulatory requirements.

Rett Syndrome Research Trust's Genetic Medicines Summit Sets the Stage for Accelerated Therapeutic Development

Retrieved on: 
Mittwoch, Oktober 11, 2023
Office of Rare Diseases Research, Food, Doctor of Philosophy, Biomarker, National Center for Advancing Translational Sciences, RSRT, Critical Path Institute, RNA, Harvard University, MD, DNA, Therapy, Summit, Center for Biologics Evaluation and Research, MPH, Roger Hawkins (drummer), IND, MacNamara, JD, Biotechnology, P.C, Rare, United States Department of the Army, MECP2, EEG, FDA, Broad Institute, Hyman, MIT, RNA editing, Clinical trial, Wuhan Institute of Biological Products, Bangladesh Technical Education Board, Regulation of tobacco by the U.S. Food and Drug Administration, COA, Rett syndrome, Pharmaceutical industry, Medical imaging, Rett, Research

TRUMBULL, Conn., Oct. 11, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts. This landmark event gathered distinguished scientists, clinicians, regulatory experts, and over 40 leading biopharma companies. The goal of the Summit was to foster dialogue among scientific, clinical, industry, and regulatory stakeholders to facilitate collaboration, identify best practices, and refine expectations to expedite pre-clinical and clinical development programs focused on genetic medicines for children and adults with Rett syndrome.

Key Points: 
  • The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts.
  • TRUMBULL, Conn., Oct. 11, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts.
  • RSRT's Rett Syndrome Genetic Medicines Summit underscores the organization's unwavering commitment to curing Rett syndrome.
  • RSRT remains steadfast in its mission to accelerate the development of genetic therapies and ultimately find a cure for Rett syndrome.

Rett Syndrome Research Trust Awards $1.1 Million to Emerald Innovations to Develop Novel Invisible Biosensor for Objective Measures of Rett Symptoms

Retrieved on: 
Dienstag, Februar 21, 2023
Emerald, Quantum Artificial Intelligence Lab, National academy, National Academy of Engineering, Artificial intelligence, Sleep, MIT, Wireless network, Therapy, Alzheimer's disease, MacArthur Fellows Program, Publication, Diagnosis, Massachusetts Institute of Technology, Drug development, Science, Radio, Patient, Computer science, Rett syndrome, Research, Sloan Research Fellowship, Privacy, Risk, Movement, Mobile computing, RSRT, Vital signs, American Academy, Physiology, FDA, American Academy of Arts and Sciences, MIT Center for Collective Intelligence, ACM, Prognosis, Network, Parkinson's disease, Environment, Doctor of Philosophy, Pharmaceutical industry, Medical imaging, Trustee, Rett, Engineering

TRUMBULL, Ct., Feb. 21, 2023 Feb.21, 2023 -- The Rett Syndrome Research Trust (RSRT) is excited to announce a $1.1 million dollar research award to Dina Katabi, PhD, and her team at Emerald Innovations. The funding will enable Dr. Katabi and Emerald Innovations to continue developing cutting-edge technology to directly and objectively assess symptoms of irregular breathing, sleep disruptions, and movement in individuals with Rett syndrome. The new study builds upon pilot results that demonstrated Emerald's feasibility to assess Rett syndrome symptoms at home, capturing significant disruptions in sleep and breathing over a four-week period — all without touching the patient.

Key Points: 
  • The Rett Syndrome Research Trust (RSRT) is excited to announce a $1.1 million dollar research award to Dina Katabi, PhD, and her team at Emerald Innovations.
  • The funding will enable Dr. Katabi and Emerald Innovations to continue developing cutting-edge technology to directly and objectively assess symptoms of irregular breathing, sleep disruptions, and movement in individuals with Rett syndrome.
  • TRUMBULL, Ct., Feb. 21, 2023 Feb.21, 2023 -- The Rett Syndrome Research Trust (RSRT) is excited to announce a $1.1 million dollar research award to Dina Katabi, PhD, and her team at Emerald Innovations.
  • The funding will enable Dr. Katabi and Emerald Innovations to continue developing cutting-edge technology to directly and objectively assess symptoms of irregular breathing, sleep disruptions, and movement in individuals with Rett syndrome.

The Rett Syndrome Research Trust Funds Herophilus’ Rett Syndrome Lead Candidate Studies

Retrieved on: 
Donnerstag, Januar 26, 2023
Health, Genetics, General Health, Research, Science, Pharmaceutical, Biotechnology, Organoid, MECP2 duplication syndrome, Therapy, Neuron, MECP2, Safety, Disorder, Myenteric plexus, Patient, Rett syndrome, Astrocyte, Risk, RSRT, Disease, Psychiatry, Machine learning, Drug discovery, Doctor of Philosophy, Pharmaceutical industry, Medical device, Rett, Herophilos

Herophilus announced today that in vivo studies underway of its lead candidate HRP-12975 are funded in part by the Rett Syndrome Research Trust (RSRT).

Key Points: 
  • Herophilus announced today that in vivo studies underway of its lead candidate HRP-12975 are funded in part by the Rett Syndrome Research Trust (RSRT).
  • HRP-12975 is the first small molecule therapy for Rett Syndrome with the potential to reverse the root cause of the disease, MECP2 deficiency.
  • With the support of RSRT, Herophilus is now generating efficacy and safety data of HRP-12975 using genetic mouse models of Rett Syndrome.
  • Reactivation of the silent MECP2 gene is an important therapeutic strategy for us and we are encouraged by Herophilus’ progress,” said Monica Coenraads, CEO of the Rett Syndrome Research Trust.