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 ...

The Bull Case For Denali Therapeutics (DNLI) Could Change Following NEJM Tividenofusp Data Publication And FDA Review

Denali Therapeutics announced that results from its open-label Phase 1/2 trial of tividenofusp alfa for Hunter syndrome were published in the January 1, 2026 issue of The New England Journal of ...
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.
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 ...

AskBio Announces FDA Acceptance of Investigational New Drug (IND) Application for AB-1009 Gene Therapy for Treatment of Late-Onset Pompe Disease (LOPD)

AB-1009 program advances to Phase 1/Phase 2; clinical trial initiated in the United StatesTherapy granted United States Food ...
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 ...
MedCity News

Two for two: Sanofi snares twin European nods for enzyme replacement therapies

It’s a big day for Sanofi as the pharmaceutical giant welcomes two European drug approvals, both for rare enzyme deficiencies. One regulatory nod ushers in the first treatment in Europe for a ...
FiercePharma

Sanofi racks up 2 approvals in Europe for enzyme replacement therapies for rare diseases

When CEO Paul Hudson took over at Sanofi in 2019, he launched a “play to win” strategy centered on first-in-class and best-in-class drugs in therapeutic areas where the company was lacking. On Tuesday ...
MedPage Today

Promising Results in First Test of In Utero Enzyme-Replacement Therapy

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The first test ...
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 ...
MedPage Today on MSN

Turning Point for Hunter Syndrome?

The devastating consequences of mucopolysaccharidosis II (MPS II), also called Hunter syndrome, may soon become preventable ...
News Medical

How has the COVID-19 pandemic impacted prescribing pancreatic enzyme replacement to people with unresectable pancreatic cancer?

In a recent study posted to the medRxiv* preprint server, researchers evaluated the impact of the coronavirus disease 2019 (COVID-19) pandemic on the rates of pancreatic enzyme replacement therapy ...