Hyper-Genetics -

Fighting Neuromuscular Diseases, one disease at a time



Become an Investor

CALL US TODAY

Hyper-Genetics -

Fighting Neuromuscular Diseases, one disease at a time



Become an Investor

CALL US TODAY

University of Florida College of Medicine Duchenne Muscular Dystrophy Clinical Trial - Supported by JAR of Hope & RESTEM, LLC


Philanthropic support has been critical to the center’s advancement of clinical trials. By partnering with the JAR of Hope Foundation and RESTEM LLC, the CRM will be able to initiate an important trial for Duchenne Muscular Dystrophy (DMD). UF has already been selected as the clinical trial site and RESTEM, LLC has received authorization from its Investigational New Drug Application to the FDA entitled “A phase 1, randomized, double-blind, placebo-controlled Cross Over study to assess the safety, tolerability, and preliminary efficacy of Umbilical Cord Lining stem cells (ULSC) in Pediatric patients with duchenne’s muscular dystrophy (DMD)” to proceed with a Phase 1 trial in 12 patients.


Additional support from the JAR of Hope Foundation will help translate Phase 1 and later Phase 2 trial with more answers as to if this will help these children with this debilitating disease. Applying the CRM’s research broadly, center leadership is seeing positive early results indicating that regenerative medicine might be a better course of treatment for some patients than immunosuppressive medicines with powerful side effects.

Click Here for Clinical Trial Information Supported by JAR of Hope

Hyper-Genetics -

Fighting Neuromuscular Diseases, one disease at a time


Become an Investor

CALL US TODAY

Our Mission is to push science forward for those who need it today

Hyper-Genetics is dedicated to moving science forward for ongoing research in the neuromuscular space. Our goal is first to provide therapies for the improvement of the patients’ lives now while trying to find a cure. Hyper-Genetics was founded and is run by Jim Raffone. Jim firmly feels through collaboration and necessity that cures can be found.

Our Ongoing Battles

What is Duchenne muscular dystrophy (DMD)?

Duchenne muscular dystrophy, also known as DMD or Duchenne, is a rare genetic disease. It primarily affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and is eventually fatal. The genetic change that causes Duchenne — a mutation in the DMD gene — happens before birth and can be inherited, or new mutations in the gene can occur spontaneously.

Source: https://www.duchenne.com/about-duchenne

What is the Cause of Duchenne?

Duchenne is caused by a genetic mutation that prevents the body from producing dystrophin, a protein that muscles need to work properly. Without dystrophin, muscle cells become damaged and weaken. Over time, children with Duchenne will develop problems walking and breathing, and eventually the muscles that help them breathe and the heart will stop working. Duchenne is an irreversible, progressive disease. There is currently no cure for Duchenne.

DMD has many variations, which makes finding a cure extremely challenging. The mission of Hyper-Genetics is to bring awareness and raise funds to enhance treatment, and ultimately to find a cure.

Source: https://www.duchenne.com/about-duchenne

The Why Hyper-Genetics is fighting Duchenne muscular dystrophy

The Hope Center: Bringing Hope to Families Who Need It

“ I want to thank JAR of Hope for their commitment and support for my son. JAR914 treatment is very beneficial to my son. Not only is he more stable and falls less frequently then before, but he has also improved upper arm and shoulder strength. Boys with Duchenne do not improve at my sons age(almost 8)devastatingly they decline each month, but JAR of Hope and our faith in God has given us just that, HOPE. We are beyond grateful and stand with JAR of Hope and their commitment to finding a cure and an effective treatment option for not only My son and Jamsey but all the beautiful boys who deserve to live a long and healthy life . We truly believe God was the one who opened this door and led us to JAR of Hope. Together we can raise awareness and move closer each day to finding a cure!”

Alina S

Mom of one of the boys in the study

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