Myotonic Dystrophy Type 2
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Learn About Myotonic Dystrophy Type 2

What is the definition of Myotonic Dystrophy Type 2?
Myotonic dystrophy type 2, one of the two types of myotonic dystrophy, is an inherited muscular dystrophy that affects the muscles and other body systems (e.g., heart, eyes, and pancreas). It is characterized by prolonged muscle tensing (myotonia) as well as muscle weakness, pain, and stiffness. Signs and symptoms usually develop during a person's twenties or thirties. Muscles in the neck, fingers, elbows, and hips are typically affected; facial and ankle muscles are less commonly involved. The severity of Myotonic dystrophy type 2 varies widely among affected people, even among family members. It is inherited in an autosomal dominant pattern and is caused by genetic changes in the CNBP gene.
What are the alternative names for Myotonic Dystrophy Type 2?
  • Myotonic dystrophy type 2
  • DM2
  • Dystrophia myotonica type 2
  • Myotonic myopathy, proximal
  • PROMM
  • Proximal myotonic myopathy
  • Ricker syndrome
Who are the top Myotonic Dystrophy Type 2 Local Doctors?
Elite in Myotonic Dystrophy Type 2
Elite in Myotonic Dystrophy Type 2

Office

355 W 16th St Ste 3200, 
Indianapolis, IN 
Languages Spoken:
English

Laurie Gutmann is a Neurologist in Indianapolis, Indiana. Dr. Gutmann is rated as an Elite provider by MediFind in the treatment of Myotonic Dystrophy Type 2. Her top areas of expertise are Paramyotonia Congenita, Myotonic Dystrophy Type 2, Myotonic Dystrophy, and Charcot-Marie-Tooth Disease.

Elite in Myotonic Dystrophy Type 2
Elite in Myotonic Dystrophy Type 2

Penn Neuroscience Center - Neurology

330 South 9th Street, 3rd Floor, 
Philadelphia, PA 
Languages Spoken:
English
Accepting New Patients

Lauren Elman is a Neurologist in Philadelphia, Pennsylvania. Dr. Elman is rated as an Elite provider by MediFind in the treatment of Myotonic Dystrophy Type 2. Her top areas of expertise are Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's Disease), Primary Lateral Sclerosis, Myotonic Dystrophy, and Myotonic Dystrophy Type 2. Dr. Elman is currently accepting new patients.

 
 
 
 
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Jeffrey M. Statland
Elite in Myotonic Dystrophy Type 2
Elite in Myotonic Dystrophy Type 2

Clinical Research Center

4350 Shawnee Mission Parkway, 
Fairway, KS 
Experience:
21+ years
Languages Spoken:
English
Accepting New Patients
Offers Telehealth

Jeffrey Statland is a Neurologist in Fairway, Kansas. Dr. Statland has been practicing medicine for over 21 years and is rated as an Elite provider by MediFind in the treatment of Myotonic Dystrophy Type 2. His top areas of expertise are Facioscapulohumeral Muscular Dystrophy (FSHD), Paramyotonia Congenita, Primary Lateral Sclerosis, Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's Disease), and Gastrostomy. Dr. Statland is currently accepting new patients.

What are the latest Myotonic Dystrophy Type 2 Clinical Trials?
A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1

Summary: The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo. There will be a single-ascending dose part of the trial and a multiple-ascending dose part. In the single-ascending dose, participants...

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A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients With Myotonic Dystrophy Type 1 and Type 2 (Phase 3)

Summary: A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES study)

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Published Date: May 02, 2022
Published By: Genetic and Rare Diseases Informnation Center