upcoming events
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Tue01Jun2021Sat05Jun2021Scottish Event Campus (SEC) in Glasgow, United Kingdom.
EPNS
learn about the latest developments in the rapidly evolving field of child neurology including basic science, diagnostic methods and novel treatments.
https://epns-congress.com/
Latest news
Interactions with FDA anticipated by mid-2021 to determine the BLA filing strategy for OTL-200 BOSTON and LONDON, Jan. 14, 2021 (GLOBE NEWSWIRE) — Orchard Therapeutics,…
Press Release: Orchard Therapeutics BOSTON/LONDON/MILAN, December 21, 2020 – Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon…
Thank you ALL for supporting last year’s sign on letter to add language to Newborn Screening Saves Lives Reauthorization which would fund states in order…
The novel coronavirus pandemic can have a serious impact on people with rare diseases and their families. Yet, not much is known about it. We…
NORD invites you to register for an externally-led Patient-Focused Drug Development (EL- PFDD) virtual meeting on Krabbe disease, taking place Thursday, October 29, 12:30 – 4:30 pm…
from BlueCross BlueShield of Illinois Look around and you’ll see caregivers. In the U.S., 1 in 3 adults give care to others. Caregivers may be…
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Dylan was born on April 15, 2009, Daddy’s Little tax Deduction! For the first 5 months he progressed like a normal child, until one day,…
From PatientWorthy.com After coming back from the Global Genes conference, Kristie was convinced that establishing a foundation was the right next step to advocate for…
“Until February 2013, our 2-year-old son Sam was completely normal child, no problems with his pregnancy or birth, he met every milestone, and overall was…