Medscape

Pancreatic Enzyme Replacement Therapy for GLP-1 Symptoms?

The therapy shows efficacy in improving diarrhea, abdominal pain in GLP-1, GLP-1/GIP-treated patients with diabetes and/or obesity in a small study.
The University of Alabama at Birmingham

Investigational gene therapy trial for rare kidney disease begins clinical trial at UAB

UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...
BioSpace

Denali’s Hunter Syndrome Candidate in the Spotlight After REGENXBIO Rejection

The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO's investigational gene therapy, ...

Lysosomal Storage Disorders: How These Rare Diseases Cause Progressive Organ Damage?

Lysosomal Storage Disorders are genetic conditions caused by enzyme deficiencies that lead to harmful substance buildup in cells, resulting in progressive organ damage. Common disorders in India ...
Healio

Enzyme replacement therapy curbs bleeding events in congenital blood clotting disorder

Please provide your email address to receive an email when new articles are posted on . Mean maximum ADAMTS13 activity after recombinant ADAMTS13 exceeded 100%. Markedly fewer patients receiving ...
MedCity News

BioMarin’s $270M Inozyme Acquisition Brings Drug That Could Become First for a Rare Disease

BioMarin Pharmaceutical is building up its pipeline with the acquisition of Inozyme Pharma in a $270 million deal centered on an enzyme replacement therapy that could become the first FDA-approved ...
WebMD

Understanding Galafold: How This Oral Treatment Works for Fabry Disease

If you or someone you know has Fabry disease, you may have heard of Galafold. This is the first and only medicine for Fabry disease that can be taken by mouth. Other treatments for Fabry disease, ...
Medscape

Exocrine Pancreatic Insufficiency: Optimal PERT Dose Varies by Primary Pancreatic Disease

VIENNA, Austria — The appropriate dose of pancreatic enzyme replacement therapy (PERT) for exocrine pancreatic insufficiency (EPI) depends on the root cause of the insufficiency, according to results ...
Everyday Health

Enzyme Therapy for Diabetes: Who Really Needs It?

"There are three digestive enzymes made by your pancreas," says Mary Vouyiouklis Kellis, MD, a specialist in the department of endocrinology, diabetes and metabolism at the Cleveland Clinic. "Most ...
Nasdaq

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 ...
News Medical

New gene therapy shows promise for lifelong treatment of hypophosphatasia

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week. It's been a ...