L'Associazione Nazionale Tay-Sachs e Malattie Alleate ospita il primo incontro del suo genere sullo sviluppo di farmaci per GM2
Foto di Hannah Busing su Unsplash
Questo è un repost dell'articolo del 29 gennaio 2024 condiviso dalla National Tay-Sachs & Allied Diseases Association.
Boston, Mass. January 29, 2024— The National Tay-Sachs & Allied Diseases Association (NTSAD), the leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases, is hosting the first-ever, ExternallyLed Patient-Focused Drug Development Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on Thursday, February 15, 2024.
Externally-led Patient-Focused Drug Development (EL-PFDD) meetings give the U.S. Food and Drug Administration (FDA) and other key stakeholders, including medical product developers, healthcare providers, and regulatory agencies, an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during their review of a marketing application.
GM2 can affect anyone, regardless of race, gender, ethnicity, or country of origin. NTSAD alone serves hundreds of families in the U.S. and globally. To date, more than 150 people including patients, researchers, clinicians, and industry members have registered for the GM2 EL-PFDD meeting. This virtual meeting will be livestreamed via YouTube, open to the public, and free to attend. However, participants must register in advance at www.ntsad.org/gm2pfdd. The meeting begins at 9:30 a.m. Eastern on February 15, 2024, and is scheduled to end at 3 p.m. Eastern.
Nearly 30 individuals who are adults or parents of children affected by infantile, juvenile, or late-onset Tay-Sachs and Sandhoff diseases will share their stories and experiences. Patients, parents, caregivers, and bereaved family members will share their perspectives.
"Condividere la storia di mia figlia e continuare a essere la sua voce per far progredire la ricerca nella speranza di un trattamento e di una cura per la GM2, anche ora, a dodici anni dalla sua morte, è indispensabile affinché nessun'altra famiglia debba conoscere questo dolore lancinante", ha dichiarato Becky Benson.
Tra gli altri relatori figurano Jacqueline Karp, MD, U.S. FDA Center for Drug Evaluation and Research; Florian Eichler, MD, Massachusetts General Hospital; Cynthia Tifft, MD, PhD, National Institutes of Health; e Kathleen M. Flynn, NTSAD Chief Executive Officer. Saranno inoltre disponibili chiamate e sondaggi in diretta per tutti i partecipanti alla riunione. Inoltre, i partecipanti potranno inviare commenti prima, durante e 30 giorni dopo la riunione attraverso il sito web del NTSAD.
"La NTSAD è stata fondata dalle famiglie. Sono loro gli esperti di queste devastanti malattie genetiche rare e la loro voce deve essere ascoltata", ha dichiarato Kathleen M. Flynn, amministratore delegato della NTSAD. "È un onore offrire alle famiglie questa opportunità unica di condividere le loro storie e i loro punti di vista per accelerare lo sviluppo di trattamenti e fare la differenza, se non per loro stesse, ma per far sì che altre famiglie non debbano sperimentare un dolore e un'angoscia duraturi".
Tra gli sponsor dell'incontro GM2 EL-PFDD figurano Sanofi, Cure Tay-Sachs Foundation, New York Area Fund at NTSAD, Mathew Forbes Romer Foundation, Azafaros e JCR Pharmaceuticals.
Per saperne di più sulla riunione GM2 EL-PFDD, registrarsi per partecipare o inviare commenti, visitare il sito www.ntsad.org/gm2pfdd o guardare un webinar informativo su https://www.youtube.com/watch?v=tuyd26kn9wU&feature=youtu.be. Il rapporto Voice of the Patient, che raccoglierà i punti salienti della riunione, sarà disponibile quest'estate.
Informazioni su NTSAD
NTSAD is among the first and most respected patient advocacy groups and was a pioneer in advancing carrier screening to prevent rare genetic diseases. The organization supports families and individuals around the world through one-on-one professional support, mentoring and peer groups, information and resources, and its Annual Family Conference. NTSAD also advocates for patients and advances research. Since 2002, the organization has awarded more than $4.7 million in grants that have been leveraged to attract more than $30 million of investments in research. These initiatives have led to the development of potential therapies for Tay-Sachs, Canavan, GM1, and Sandhoff diseases. For more information, please visit NTSAD.org.
