The FDA approved Genglycos (pariglasgene brecaparvovec-opnr) to reduce daily cornstarch intake in patients aged 8 years and older with glycogen storage disease type Ia. Known as Von Gierke disease, GSDIa is a rare metabolic disorder caused by a mutation in the G6PC gene. This genetic variation leads to a deficiency in glucose-6-phosphatase (G6Pase), an enzyme needed to release glucose into the bloodstream. Without this enzyme, the body cannot properly maintain blood glucose levels, causing severe hypoglycemia and other serious metabolic complications · Pariglasgene brecaparvovec is an adeno-associated virus (AAV) serotype 8 based gene therapy that delivers a functional copy of the G6PC gene into liver cells, enabling the production of normally functioning G6Pase. Ultragenyx stated that as part of its postmarketing commitments to the FDA, the Company will provide 2 years of clinical data from open-label commercial treatment of 50 patients and 20 control patients through its existing GSDIa Disease Monitoring Program. ... Ultragenyx announces US FDA approval of Genglycos™ gene therapy, the first-ever FDA-approved treatment designed to treat the underlying cause of glycogen storage disease type Ia (GSDIa). “The reduced reliance on cornstarch, experienced by patients in our clinical studies, demonstrates this gene therapy’s ability to establish the normal breakdown of glycogen to produce glucose during fasting or episodes of metabolic stress. This ability to regulate glucose has alleviated the disease burden and has the potential to mitigate the risk of severe or life-threatening hypoglycemia for these patients.” Close more info about First Gene Therapy Approved for Glycogen Storage Disease Type la

Las Vegas Nevada United States As per DelveInsight s assessment globally Netherton Syndrome pipeline constitutes 5 key companies continuously working towards developing 5 Netherton Syndrome treatment therapies analysis of Clinical Trials Therapies Mechanism of Action Route of Administration ... Las Vegas Nevada United States As per DelveInsight s assessment globally Netherton Syndrome pipeline constitutes 5 key companies continuously working towards developing 5 Netherton Syndrome treatment therapies analysis of Clinical Trials Therapies Mechanism of Action Route of Administration and Developments ... This represents an important milestone as Quoin progresses its therapeutic candidate into late-stage clinical development. • In April 2025, ResVita Bio, a therapeutics company specializing in skin disease treatments, announced that the FDA has granted Orphan Drug Designation to RVB-003 for Netherton Syndrome, a serious and chronic skin disorder. Building on the FDA's earlier Rare Pediatric Disease Designation, this milestone highlights ResVita Bio's innovative continuous protein therapy platform, which delivers sustained drug levels directly to the skin, offering enhanced efficacy and improved safety compared to conventional topical treatments. • Netherton Syndrome companies working in the treatment market are Quoin Pharmaceutical, Boehringer Ingelheim, LifeMax Laboratories, Novartis, Daiichi Sankyo, Quoin Pharmaceuticals, Children's Hospital of Philadelphia, and others, are developing therapies for the Netherton Syndrome treatment • Emerging Netherton Syndrome therapies in the different phases of clinical trials are- QRX003, SPEVIGO (spesolimab/BI 655130), LM-030 (BPR277), DS-2325a, Pimecrolimus, and others are expected to have a significant impact on the Netherton Syndrome market in the coming years. • In March 2026, Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX), a late-stage specialty pharmaceutical company focused on rare and orphan diseases, announced a clinical and regulatory update following a constructive Type C meeting with the U.S. Press release - DelveInsight Business Research - Netherton Syndrome Pipeline 2026: FDA Updates, Therapy Innovations, and Clinical Trial Landscape Analysis by DelveInsight - published on openPR.com

Key neurology trials are set to report data in early 2026, including the ADEPT-2 study on xanomeline/trospium for Alzheimer's psychosis and the ELEVATE-PD trial on IPX203 for Parkinson's. These studies may introduce new therapies and impact treatment strategies for Alzheimer's and Parkinson's disease.