Immusoft Receives FDA Rare Pediatric Disease Designation for ISP-002 to treat Mucopolysaccharidosis Type II

Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) ...
Healio

Enzyme-replacement therapy improves outcomes in Pompe disease

Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.
Medical Xpress

Enzyme replacement therapy offers hope for ultra-rare Hunter syndrome

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic ...

Collaborations Pharmaceuticals, Inc. demonstrates a treatment for CLN1 Batten disease crosses the blood-brain-barrier

PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...
MarketWatch

Spruce Biosciences Receives U.S. FDA Breakthrough Therapy Designation for Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) in Sanfilippo Syndrome Type B (MPS IIIB)

Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need ...
Medical Xpress

Therapeutic or Preventive Procedure:

A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for ...
WRAL

Enzyme Replacement Therapy Being Used To Treat Hunter Syndrome

Children with Hunter Syndrome lack a certain enzyme in their blood. The Food and Drug Administration just approved an enzyme-replacement therapy for these patients. It had its start at UNC Hospitals.
FOX6 News Milwaukee on MSN

Muskego family helps raise millions for rare childhood disease treatment

A Muskego family helped raise more than $5.5 million to fund early access treatment for children with Sanfilippo syndrome ...
Monthly Prescribing Reference

Enzyme Replacement Therapy Fast Tracked for ABCC6 Deficiency

In early trial data, INZ-701 was associated with several clinical benefits including a reduction or stabilization of carotid intima-media thickness. The Food and Drug Administration (FDA) has granted ...
MedPage Today

Gene Therapy Shows Lasting Benefit for Children With Rare Disorder

An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...
Seeking Alpha

Spruce Biosciences Receives U.S. FDA Breakthrough Therapy Designation for Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) in Sanfilippo Syndrome Type B (MPS IIIB)

Breakthrough Therapy Designation Supported by Integrated Long-Term Clinical Data Demonstrating Normalization in Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE) U.S. FDA Confirmed ...