-
Next Generation Gene Therapeutics (NGGT) Announces Publication of Early Clinical Trial Results for NGGT001 for the Treatment of Bietti's Crystalline Dystrophy (BCD)
14 Jan 2025 18:01 GMT
… .
Key findings of the trial:
Safety: The trial found no substantial safety … -stage biotechnology company developing novel gene therapies for the treatment of retinal … ;II-ready gene therapy for PKU; NGGT006, a gene therapy for …
-
Chile Inborn Errors Of Protein Metabolism Market To Generate Valuation Of US$ 217.2 Million By 2032 | Phenylketonuria (PKU) Disorder Take Up Over 46% Market Share Says Astute Analytica
24 Dec 2024 18:12 GMT
… in more than 200 pharmacies nationwide, ensuring better … By Disorder Type Phenylketonuria (PKU) (46%) By Treatment Type Medical Foods … dedicated to treating metabolic disorders, with PKU being a … investments in research and development. Nestlé leads with …
-
Pikka '25 development plan and 5-yr unit term extension approved - 01/12/2025
10 Jan 2025 03:18 GMT
… Pikka Unit, or PKU. Plan of Development from Oil Search ( … , 2025. The PKU is now under development with surface construction … support installation of the Seawater Treatment Plant, or STP, facility. … six to eight development wells within the PKU.
--KAY CASHMAN …
-
iECURE Reports Complete Clinical Response in First Infant Dosed with its In Vivo Gene Editing Candidate ECUR-506 in an Ongoing Phase 1/2 Clinical Trial in Ornithine Transcarbamylase (OTC) Deficiency
09 Jan 2025 13:39 GMT
… development of mutation-agnostic in vivo gene insertion therapies for the treatment … , in future clinical trial participants.”
Summary of … of care ammonia scavenger medication and protein restriction. … also assess the pharmacokinetics and efficacy of ECUR …
-
FDA Accepts Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia
08 Jan 2025 12:00 GMT
… Drug Application (sNDA) for PYRUKYND® (mitapivat) for the treatment … a disease-modifying oral medication, to thalassemia patients … is dedicated to developing and delivering transformative … MDS)-associated anemia and phenylketonuria (PKU). In addition to its …
-
Eton Pharma acquires Galzin, an US FDA approved treatment of ultra─rare metabolic condition of Wilson disease
06 Jan 2025 09:52 GMT
… Pharmaceuticals, Inc, an innovative pharmaceutical company focused on developing and commercializing treatments … a critical medication for patients with … healthcare professionals who treat Wilson disease. … Increlex, Alkindi Sprinkle, Pku Golike, Carglumic acid …
-
Eton Pharmaceuticals Advances Its Commitment to Rare Disease with the Acquisition of Galzin®
03 Jan 2025 11:50 GMT
… innovative pharmaceutical company focused on developing and commercializing treatments for … is a critical medication for patients with … healthcare professionals who treat Wilson Disease. Once … of discovering, developing and commercializing drugs that are …
-
New hope for male infertility: PKU researchers discover key mechanism in Klinefelter syndrome
03 Jan 2025 18:11 GMT
Imagine the deep frustration of countless men who long to become fathers, only to face infertility due to a genetic condition they can't control. For those with Klinefelter syndrome, this painful reality is a constant struggle.
How does an extra X …
-
Tebapivat by Agios Pharmaceuticals for Sickle Cell Disease: Likelihood of Approval
01 Jan 2025 06:36 GMT
… historical drug development data. Attributes of the drug, … Pharmaceuticals overview
Agios Pharmaceuticals (Agios) discovers and develops investigational medicines for the treatment … stabilizer that treats phenylketonuria (PKU); mitapivat treats adult pyruvate …
-
Unveiling the Mechanisms and Therapeutic Effects of Xiaoyao Sanjie Decoction in Triple-Negative Breast Cancer: A Network Pharmacology and Experimental Validation Approach
27 Dec 2024 08:22 GMT
… causes of tumor occurrence and development.21,22 Therefore, on … of XYSJD treatment for TNBC remains unclear.
Network pharmacology establishes a … for 24 h before drug treatment. The transfection efficiency was … Que is effective in treating TNBC both in vitro …