Pompe Disease: How This Rare Genetic Disorder Causes Progressive Muscle Weakness and Respiratory Failure

On World Rare Disease Day, we take a look at Pompe disease - a rare genetic disorder caused by a deficiency of the GAA enzyme, leading to glycogen buildup in muscles. It results in progressive muscle ...

U.S. FDA has granted accelerated approval of Loargys(R) (pegzilarginase-nbln) for the treatment of hyperargininemia in patients 2 years and older with Arginase 1 Deficiency ...

LOARGYS is the first and only therapy proven to lower arginine in patients 2 years of age and older living with ARG1-D -- ARG1-D is an ultra-rare, debilitating, and progressive metabolic disease ...

U.S. FDA has granted accelerated approval of Loargys® (pegzilarginase-nbln) for the treatment of hyperargininemia in patients 2 years and older with Arginase 1 Deficiency (ARG1-D)

Immedica Pharma today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Loargys® (pegzilarginase-nbln), an arginine specific enzyme indicated for the ...
Endocrinology Advisor

FDA Grants Accelerated Approval to Loargys for Arginase 1 Deficiency

Phase 3 PEACE trial data support the accelerated approval of Loargys for the treatment of arginase 1 deficiency in pediatric and adult patients.
Fierce Pharma

Immedica overcomes prior FDA rebuke for ultrarare disease med to score new approval

Four years after the FDA issued its most heavy-handed form of a rejection to the prior company behind pegzilarginase, the U.S. regulator has now given the treatment a thumbs-up. | Four years after the ...
BioSpace

Rare Disease Groups Lobby for Regulatory Expediency as Makary Defends ‘Rigorous’ Approach

This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite ...
pharmaphorum

NICE backs first disease-modifying drug for ARG1 deficiency

An enzyme replacement therapy developed by Immedica, the first drug for the inherited metabolic disorder arginase-1 (ARG1) deficiency that can alter the course of the disease, has been cleared for NHS ...

FDA moves to facilitate personalized therapies for rare diseases

The Food and Drug Administration is making it possible for pharmaceutical companies to produce bespoke medicines for individual patients, an effort to revolutionize the standard of care for rare ...
TheHealthSite on MSN

Iron Deficiency And Hair Loss: Why This Common Nutrient Shortage Causes Hair Thinning

Insufficient iron supply to your body can prevent haemoglobin production which can repair cells in the body, including ...

Brain supplements have become a social media wellness trend - but are they safe to take?

On social media, boosting brain health has become a wellness trend - and influencers point to taking fish oil and other ...

Utah adding to newborn screening. What can the testing find?

Mandatory testing of 45 genetic disorders is designed to lead to treatment before symptoms do damage.

Denali Therapeutics Presents Enzyme TransportVehicle™ Progress Across Three Clinical Programs for Treatment of Lysosomal Storage Disorders at 2026 WORLDSymposium™

Analysis from continued follow-up of Phase 1/2 clinical trial data in Hunter syndrome (MPS II) reinforces potential for tividenofusp alfa ...