NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because ... Read more
Updates
-
-
FLT190 Granted Orphan Drug Status in Europe as Potential Gene Therapy for Fabry Disease
The gene therapy candidate FLT190 — designed to increase blood levels of the enzyme alpha-GAL A, which is affected in Fabry disease — has ... Read more -
For My Daughter on Her Birthday
I’m writing this column on the eve of my daughter’s 13th birthday. We are six days into social distancing in an effort to help ... Read more -
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based ... Read more -
Newborn Screening ‘Best Way’ to Diagnose Fabry in Infants, Family Members, Study Suggests
Newborn screening programs are the best way to diagnose Fabry disease early in infancy — enabling timely enzyme replacement therapy before the disease causes ... Read more
-
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, ... -
Travel Tips for People Affected by Fabry Disease
Fabry disease is a rare genetic disease that results from abnormal buildup of a ... -
In Exercise and Health, We Keep Moving Forward
Feb. 29 wasn’t just leap day, it also was Rare Disease Day. I wrote in ... -
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media ... -
Warning Signs of Depression in Fabry Disease Patients
Fabry disease is a rare genetic disease in which mutations in the GLA gene ... -
PRX-102 Leads to Improvement in Fabry Patients, Phase 1/2 Data Suggest
PRX-102 (pegunigalsidase alfa), Protalix BioTherapeutics’ investigational enzyme replacement therapy, led to clinical improvements in ...
Subscribe to our mailing list
Get regular updates delivered to your email box.
Thanks! Please check your email for a confirmation email.
Protalix’s Pegunigalsidase Alfa Granted FDA Fast Track Status for Fabry Disease
News
The U.S. Food and Drug Administration (FDA) has granted fast track designation to pegunigalsidase alfa, or PRX-102, Protalix BioTherapeutics’ therapy candidate under development for Fabry disease. FDA’s fast track status is designed to facilitate the development and speed up FDA review ... Read more