-
Maze Therapeutics Announces Positive First-in-Human Results from Phase 1 Trial of MZE782, Establishing Proof of Mechanism for a Potent, Oral SLC6A19 Inhibitor with Potential to Treat Phenylketonuria (PKU) and Chronic Kidney Disease (CKD)
11 Sep 2025 11:30 GMT
… the potential to treat both phenylketonuria (PKU) and chronic kidney disease …
-
BioMarin Announces Positive Pivotal Phase 3 Data for PALYNZIQ® (pegvaliase-pqpz) in Adolescents with Phenylketonuria at 15th International Congress of Inborn Errors of Metabolism
06 Sep 2025 15:29 GMT
… of Pegvaliase in Adolescents with Phenylketonuria: Primary Results from PEGASUS, … Desensitization Protocol for Adults with Phenylketonuria Experiencing Hypersensitivity Reactions to … -pqpz-in-adolescents-with-phenylketonuria-at-15th-international-congress- …
-
Phenylketonuria Pipeline Outlook 2025: Clinical Trial Studies, EMA, PDMA, FDA Approvals, MOA, ROA, NDA, IND, And Companies
23 Aug 2025 02:47 GMT
… .....
Inactive Products
Phenylketonuria Key Companies
Phenylketonuria Key Products
Phenylketonuria- Unmet Needs
Phenylketonuria- Market Drivers …
-
US FDA approves PTC Therapeutics’ Sephience to treat children and adults living with phenylketonuria
30 Jul 2025 09:29 GMT
… children and adults living with phenylketonuria (PKU). The approval includes broad … adult and paediatric patients with phenylketonuria (PKU). Sephience is a natural … a phenylalanine (Phe)-restricted diet.
Phenylketonuria (PKU) is a rare, inherited …
-
PTC’s Sephience wins FDA nod for phenylketonuria
30 Jul 2025 01:30 GMT
… . will be launching its oral phenylketonuria therapy, Sephience (sepiapterin) in both …
-
Phenylketonuria Pipeline Outlook Report 2025: Key 20+ Companies And Breakthrough Therapies Shaping The Future Landscape
29 Jul 2025 00:25 GMT
… .....
Inactive Products
Phenylketonuria Key Companies
Phenylketonuria Key Products
Phenylketonuria- Unmet Needs
Phenylketonuria- Market Drivers …
-
FDA approves sepiapterin to treat phenylketonuria in adult, pediatric patients
29 Jul 2025 01:36 GMT
… adult and pediatric patients with phenylketonuria (PKU), including all age groups … Children and Adults Living with Phenylketonuria (PKU). PTC Therapeutics. Press release …
-
FDA Approves Sepiapterin for Adults, Children Living With Phenylketonuria
28 Jul 2025 23:05 GMT
… adults and children living with phenylketonuria (PKU), a rare, inherited … Children and Adults Living with Phenylketonuria (PKU). News Release. Released April … of PTC923 in Participants With Phenylketonuria. National Library of Medicine. ClinicalTrials …
-
PTC Therapeutics Announces FDA Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
28 Jul 2025 21:19 GMT
… children and adults living with phenylketonuria (PKU). The approval includes broad … adult and pediatric patients with phenylketonuria (PKU). Sephience is a … see Full Prescribing Information.
About Phenylketonuria
Phenylketonuria (PKU) is a rare, inherited …
-
Rare Disease Show - Episode 25: Understanding Phenylketonuria ft Dr Roshan Daniel, KIMSHEALTH
15 Jul 2025 21:59 GMT
… ;s show we will cover Phenylketonuria.
Phenylketonuria (PKU) is a rare inherited … is the genetic basis of Phenylketonuria, and how is it inherited …
About
© 1995-2025