"2019 is off to a strong start as we advance our pipeline of gene therapies for lysosomal storage diseases
on multiple fronts.
The plato platform is designed to enhance the potency, safety, efficacy, and long-term durability of Avrobio's gene therapies, and may additionally provide the capability to address central nervous system manifestations that accompany many lysosomal storage diseases
As with many lysosomal storage diseases
, initiation of treatment before the onset of irreversible symptoms may be advantageous.
Lysosomal Storage diseases
(LSD), which are related to a deficiency of specific lysosomal hydrolases, resulted in clinical aspects due to an accumulation of substrates in different tissues.
The money is for the MPS Society, who support families of people with any of the 25 rare lysosomal storage diseases
, so that research might benefit Isla as she grows.
Hopwood, "Epidemiology of lysosomal storage diseases
: an overview," in Fabry Disease: Perspectives from 5 Years of FOS, A.
FDA for lysosomal storage diseases
and is developing FINDER, a rapid near-patient newborn testing solution.
The lysosomal storage diseases
are a group of rare, inherited metabolic diseases affecting about 1 in 7000 to 8000 people.
It is progressing earlier stage programmes for lysosomal storage diseases
, including next-generation therapeutics for NPC, a rare genetic disorder.
In several lysosomal storage diseases
, the activity of GCase is reduced due to genetic mutations in the GBA1 gene.
The severe neonatal form of MPS-VII is thus among the very few lysosomal storage diseases
that might present as early as at birth ; it could even be detected in utero (although unfortunately often missed ).