Acute hepatic porphyria

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ORPHA:95157
Who is this for?
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1FDA treatments2Active trials45Specialists8Treatment centers1Financial resources

Where are you in your journey?

ℹ
UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Orphanet ↗NORD ↗

FDA & Trial Timeline

5 events
Jan 2024Clinical Research on Acute Intermittent Porphyria and the Use of Carbohydrate-Rich Diet as a Treatment

Nordlandssykehuset HF — NA

TrialRECRUITING
Jan 2022Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome

Vanderbilt University Medical Center

TrialACTIVE NOT RECRUITING
Apr 2021ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

Alnylam Pharmaceuticals

TrialRECRUITING
Nov 2019

GIVLAARI: FDA approved

GIVLAARI is indicated for the treatment of adults with acute hepatic porphyria (AHP).

FDAcompleted
Nov 2010Longitudinal Study of the Porphyrias

Icahn School of Medicine at Mount Sinai

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

GIVLAARI

givosiran· Alnylam Pharmaceuticals, Inc.Orphan Drug

GIVLAARI is indicated for the treatment of adults with acute hepatic porphyria (AHP).

View Details →FDA Label ↗Copay Card ↗Patient Assistance ↗

Clinical Trials

2 recruitingView all trials with filters →
Other2 trials
Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome
Active
PI: Cyndya Shibao, M.D (Vanderbilt University Medical Center) · Sites: Nashville, Tennessee · Age: 18–65 yrs
View on ClinicalTrials.gov ↗I'm interested →
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
Actively Recruiting
PI: Medical Director (Alnylam Pharmaceuticals) · Sites: Birmingham, Alabama; Gilbert, Arizona +28 more · Age: 12–99 yrs
View on ClinicalTrials.gov ↗I'm interested →

Specialists

Showing 25 of 45View all specialists →
SM
Stephen Meninger
Specialist
6 Acute hepatic porphyria publications
PV
Paolo Ventura
Specialist
5 Acute hepatic porphyria publications
DV
Daphne Vassiliou
Specialist
5 Acute hepatic porphyria publications
BW
Bruce Wang
SAN FRANCISCO, CA
Specialist
4 Acute hepatic porphyria publications
HB
Herbert L Bonkovsky
WINSTON SALEM, NC
Specialist
7 Acute hepatic porphyria publications
IF
Igor Braga Farias
Specialist
3 Acute hepatic porphyria publications
BB
Bruno de Mattos Lombardi Badia
Specialist
3 Acute hepatic porphyria publications
MT
Manish Thapar
PHILADELPHIA, PA
Specialist
4 Acute hepatic porphyria publications
KS
Katharina Schmolly
LOS ANGELES, CA
Specialist
4 Acute hepatic porphyria publications
MB
Manisha Balwani
NEW YORK, NY
Specialist
4 Acute hepatic porphyria publications
MS
Marianne T Sweetser
Specialist
5 Acute hepatic porphyria publications
ES
Eliane Sardh
Specialist
7 Acute hepatic porphyria publications
KM
Karl E Anderson, MD
Specialist
PI on 3 active trials
EP
Eliane Sardh, MD PhD
Specialist
PI on 1 active trial
KA
Karl E Anderson
Specialist
PI on 2 active trials
NM
Nader Najafian, MD
BOSTON, MA
Specialist
PI on 3 active trials
CM
Christer Andersson, MD
Specialist
PI on 1 active trial
CM
Cyndya Shibao, M.D
Specialist
PI on 4 active trials
EP
Elin Storjord, MD PhD
Specialist
PI on 1 active trial
MM
Manisha C Balwani, MD
NEW YORK, NY
Specialist
PI on 1 active trial
PP
Per Dahlqvist, MD PhD
Specialist
PI on 1 active trial
DF
Dr. Yolandi Swart, FCPHM(SA)
Specialist
PI on 1 active trial
HM
Herbert L. Bonkovsky, M.D.
WINSTON SALEM, NC
Specialist
PI on 1 active trial
KM
Krzysztof Jaworski, MD
Specialist
PI on 1 active trial
JM
Joseph R. Bloomer, M.D.
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center ↗

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center ↗

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program ↗

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site ↗

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site ↗

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site ↗

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine ↗

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program ↗

UCLA Health

📍 Los Angeles, CA

Financial Resources

1 resources
GIVLAARI(givosiran)— Alnylam Pharmaceuticals, Inc.

Travel Grants

No travel grants are currently matched to Acute hepatic porphyria.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Acute hepatic porphyria

Disease timeline:

New recruiting trial: Longitudinal Study of the Porphyrias

A new clinical trial is recruiting patients for Acute hepatic porphyria

New recruiting trial: Identification of Acute Intermittent Porphyria Modifying Genes

A new clinical trial is recruiting patients for Acute hepatic porphyria

New recruiting trial: ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

A new clinical trial is recruiting patients for Acute hepatic porphyria

New recruiting trial: Clinical Research on Acute Intermittent Porphyria and the Use of Carbohydrate-Rich Diet as a Treatment

A new clinical trial is recruiting patients for Acute hepatic porphyria

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Acute hepatic porphyria

Are there clinical trials for Acute hepatic porphyria?

Yes — 2 recruiting clinical trials are currently listed for Acute hepatic porphyria on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Acute hepatic porphyria?

25 specialists and care centers treating Acute hepatic porphyria are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.