Congenital Malformation Syndrome

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2FDA treatments28Specialists8Treatment centers

Where are you in your journey?

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

Congenital Malformation Syndrome is treated with 2 medications in our database, including VIJOICE, Proteus. 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Novartis. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Congenital Malformation Syndrome treatment below.

NORD ↗

FDA & Trial Timeline

10 events
May 2026Velopharyngeal Insufficiency After Maxillomandibular Advancement Osteotomy in Obstructive Sleep Apnea Patients

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

TrialNOT YET RECRUITING
Sep 2025Multicenter Study of Patients With SHANK3 Mutations: Identification of Genes Modificators in Phelan-McDermid Syndrome (EUQ13)

Assistance Publique - Hôpitaux de Paris — NA

TrialNOT YET RECRUITING
Feb 2025Gene and Molecular Pathways of Ozone Treatment Response in Gynecological Tumor Patients With Chronic Pelvic Pain Secondary to Cancer Treatment

Bernardino Clavo, MD, PhD

TrialRECRUITING
Feb 2025Artificial Intelligence-assisted Colonoscopy in the Detection and Characterization of Colorectal Lesions

Instituto do Cancer do Estado de São Paulo — NA

TrialRECRUITING
Mar 2024A Randomized Controlled Trial Comparing Fat-free Versus Balanced (WHO) Diet in Gallstone Disease

University Hospital of Crete — NA

TrialRECRUITING
Jan 2024The Co-Op @ HeartWorks

HeartWorks, Inc.

TrialRECRUITING
Dec 2023Strategy for Management of Patients With Hereditary Cancer Syndromes (HCS) in a Rural Environment

University of Vermont Medical Center — NA

TrialENROLLING BY INVITATION
Dec 2023Multifidus Cervicis Plane Block Vs. Sham Block For Posterior Cervical Spine Fusion Surgery

Hartford Hospital — PHASE4

TrialRECRUITING
Nov 2023Discrimination of Right Versus Left Septal Accessory Pathway Before and During the Electrophysiological Study

Assiut University

TrialRECRUITING
Sep 2023Treatment Result of Zirconia Laminate Veneer

Hanoi Medical University — NA

TrialRECRUITING

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

Treatments

2 available

VIJOICE

alpelisib· Novartis

* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Not Publicly Available * RxBIN, PCN, and Group numbers: Not Publi

Proteus

ACTIVATED CHARCOAL - AETHUSA CYNAPIUM - ALTHAEA OFFICINALIS LEAF - BILBERRY - CANDIDA ALBICANS - UNCARIA TOMENTOSA - ESCHERICHIA COLI - INDOLE - MERCURIUS SOLUBILIS - OKOUBAKA AUBREVILLEI BARK - PORK · Proteus Digital Health

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

No actively recruiting trials found for Congenital Malformation Syndrome at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Congenital Malformation Syndrome community →

Specialists

Showing 25 of 28View all specialists →
MR
Małgorzata Rydzanicz
Specialist
1 Congenital Malformation Syndrome publication
&R
Śmigiel Robert
Specialist
1 Congenital Malformation Syndrome publication
JP
Justyna Paprocka
Specialist
1 Congenital Malformation Syndrome publication
MN
Magdalena Nowak
SPRINGFIELD, MA
Specialist
1 Congenital Malformation Syndrome publication
MN
Maria Nieć
Specialist
1 Congenital Malformation Syndrome publication
IJ
Izabela Janik
Specialist
1 Congenital Malformation Syndrome publication
AJ
Aleksandra Jezela-Stanek
Specialist
1 Congenital Malformation Syndrome publication
RP
Rafał Płoski
Specialist
1 Congenital Malformation Syndrome publication
KR
Karolina Rutkowska
Specialist
1 Congenital Malformation Syndrome publication
MK
Magdalena Klaniewska
Specialist
1 Congenital Malformation Syndrome publication
SS
Sivagamy Sithambaram
Specialist
1 Congenital Malformation Syndrome publication
KN
Kausthubham Neethukrishna
Specialist
1 Congenital Malformation Syndrome publication
AD
Ashwin Dalal
Specialist
1 Congenital Malformation Syndrome publication
KG
Katta Mohan Girisha
Specialist
1 Congenital Malformation Syndrome publication
TM
Tippi C MacKenzie, MD
San Francisco, California
Specialist

Rare Disease Specialist

PI on 2 active trials
FM
Forbes D Porter, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 14 active trials
AM
Amy S Paller, MD
CHICAGO, IL
Specialist
PI on 4 active trials
FM
Fanfan Hou, MD
Specialist
PI on 2 active trials
PM
Pamela A Guerrerio, M.D.
Specialist
PI on 3 active trials
JP
Jan J Koornstra, MD PhD
Specialist
PI on 2 active trials
AP
Anne-Claude TABET, MD, PhD
Specialist
PI on 2 active trials
MP
Marc Spaanderman, MD, PhD
Specialist
PI on 2 active trials
LP
Louise M O'Brien, PhD
Specialist
PI on 4 active trials
TP
Tomasz Szczapa, MD, PhD
Specialist
PI on 2 active trials

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

Travel Grants

No travel grants are currently matched to Congenital Malformation Syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Congenital Malformation SyndromeForum →

No community posts yet. Be the first to share your experience with Congenital Malformation Syndrome.

Start the conversation →

Latest news about Congenital Malformation Syndrome

Disease timeline:

New recruiting trial: Gene and Molecular Pathways of Ozone Treatment Response in Gynecological Tumor Patients With Chronic Pelvic Pain Secondary to Cancer Treatment

A new clinical trial is recruiting patients for Congenital Malformation Syndrome

New recruiting trial: Treatment Result of Zirconia Laminate Veneer

A new clinical trial is recruiting patients for Congenital Malformation Syndrome

New recruiting trial: Delineating the Molecular Spectrum and the Clinical, Imaging and Neuronal Phenotype of Chopra-Amiel-Gordon Syndrome

A new clinical trial is recruiting patients for Congenital Malformation Syndrome

New recruiting trial: Solid Tumors in RASopathies

A new clinical trial is recruiting patients for Congenital Malformation Syndrome

New recruiting trial: Effect of Music Therapy on Infants With Neonatal Abstinence Syndrome

A new clinical trial is recruiting patients for Congenital Malformation Syndrome

New recruiting trial: A Randomized Controlled Trial Comparing Fat-free Versus Balanced (WHO) Diet in Gallstone Disease

A new clinical trial is recruiting patients for Congenital Malformation Syndrome

New recruiting trial: Artificial Intelligence-assisted Colonoscopy in the Detection and Characterization of Colorectal Lesions

A new clinical trial is recruiting patients for Congenital Malformation Syndrome

New recruiting trial: Collecting Blood and Stool Samples to Detect Colorectal Cancer or Advanced Neoplasia in Lynch Syndrome Patients

A new clinical trial is recruiting patients for Congenital Malformation Syndrome

New recruiting trial: Klinefelter Syndrome and Testosterone Treatment in Puberty

A new clinical trial is recruiting patients for Congenital Malformation Syndrome

New recruiting trial: Pelvic Vein Quantitative Flow Characterization Using 2D and 4D Flow MRI

A new clinical trial is recruiting patients for Congenital Malformation Syndrome

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 Congenital Malformation Syndrome

What is Congenital Malformation Syndrome?

Congenital Malformation Syndrome is treated with 2 medications in our database, including VIJOICE, Proteus. 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Novartis. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Congenital Malformation Syndrome treatment below.

Which specialists treat Congenital Malformation Syndrome?

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

What treatment and support options exist for Congenital Malformation Syndrome?

2 FDA-approved treatments are currently tracked on UniteRare for Congenital Malformation Syndrome. See the treatments and support programs sections for copay assistance, eligibility, and contact details.