Get the up-to-date Sapropterin for BH4 deficiency Initial and continuing PBS authority application 2024 now

Get Form
Sapropterin for BH4 deficiency Initial and continuing PBS authority application Preview on Page 1

Here's how it works

01. Edit your form online
01. Edit your form online
Type text, add images, blackout confidential details, add comments, highlights and more.
02. Sign it in a few clicks
02. Sign it in a few clicks
Draw your signature, type it, upload its image, or use your mobile device as a signature pad.
03. Share your form with others
03. Share your form with others
Send it via email, link, or fax. You can also download it, export it or print it out.

The easiest way to modify Sapropterin for BH4 deficiency Initial and continuing PBS authority application in PDF format online

Form edit decoration
9.5
Ease of Setup
DocHub User Ratings on G2
9.0
Ease of Use
DocHub User Ratings on G2

Handling documents with our feature-rich and intuitive PDF editor is easy. Follow the instructions below to fill out Sapropterin for BH4 deficiency Initial and continuing PBS authority application online quickly and easily:

  1. Log in to your account. Log in with your email and password or create a free account to try the product before upgrading the subscription.
  2. Import a document. Drag and drop the file from your device or add it from other services, like Google Drive, OneDrive, Dropbox, or an external link.
  3. Edit Sapropterin for BH4 deficiency Initial and continuing PBS authority application. Effortlessly add and highlight text, insert images, checkmarks, and icons, drop new fillable areas, and rearrange or delete pages from your document.
  4. Get the Sapropterin for BH4 deficiency Initial and continuing PBS authority application completed. Download your adjusted document, export it to the cloud, print it from the editor, or share it with other participants via a Shareable link or as an email attachment.

Benefit from DocHub, the most straightforward editor to quickly manage your documentation online!

be ready to get more

Complete this form in 5 minutes or less

Get form

Got questions?

We have answers to the most popular questions from our customers. If you can't find an answer to your question, please contact us.
Contact us
Finally, in the recycling pathway, after BH4 is used as a mandatory enzyme cofactor and transformed into quinonoid dihydrobiopterin (qBH2) by pterin 4-carbinolamine dehydratase (PCD), dihydropteridine reductase (DHPR) acts on the qBH2 intermediate to regenerate BH4 [1,2] (Figure 1).
Treatment of tetrahydrobiopterin (BH4) deficiencies consists of BH4 supplementation or diet to control blood Phe and supplements of folinic acid (10-20 mg/d) in dihydropteridine reductase (DHPR) deficiency.
Sapropterin is used to lower phenylalanine levels in the blood of patients with phenylketonuria (PKU). High levels of phenylalanine (an amino acid) in the blood can cause severe brain damage, including severe mental retardation, seizures, tremors, or decreased learning ability.
Treatment of BH4 deficiencies consists of BH4 supplementation (2-20 mg/kg per day) or diet to control blood phenylalanine concentration and replacement therapy with neurotransmitters precausers (L-dopa/CarbiDOPA and 5-hydroxytryptophan), and supplements of folinic acid in DHPR deficiency.
Treatment of BH4 deficiencies consists of BH4 supplementation (2-20 mg/kg/d) or diet to control blood Phe and, in DHPR deficiency, supplements of folinic acid (10-20 mg/d).
be ready to get more

Complete this form in 5 minutes or less

Get form

People also ask

INDICATIONS AND USAGE Kuvan (sapropterin dihydrochloride) is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet.
INDICATIONS AND USAGE Kuvan (sapropterin dihydrochloride) is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU).
Rather, studies have shown that MTHF supplementation can increase BH4 levels by preventing its oxidation into BH2 and by supplementing BH4 activity with NOS enzymes further preventing its degradation.
All the people with PKU who respond positively to BH4 have at least some residual PAH enzyme activity. Sapropterin, by enhancing the activity of residual PAH enzyme, may increase tolerance to phenylalanine and allow a less restrictive diet (Blau 2004; Erlandsen 2004).
Sapropterin is used to lower phenylalanine levels in the blood of patients with phenylketonuria (PKU). High levels of phenylalanine (an amino acid) in the blood can cause severe brain damage, including severe mental retardation, seizures, tremors, or decreased learning ability.

Related links