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Treatments for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a progressive genetic condition predominantly affecting boys and men, characterized by a gradual loss of muscle function over time. Although present from birth, symptoms typically manifest in early childhood. Current treatment options for DMD focus on slowing disease progression and managing associated symptoms, such as muscle tightness, cardiovascular issues, and respiratory problems.

Steroid therapy is a primary treatment approach for DMD. Physicians often prescribe corticosteroids to patients, aiming to boost muscle strength and delay the loss of mobility by up to two to three years. Steroids also help reduce the risk of spinal curvature and enhance breathing function in individuals with DMD. Initiating steroid treatment early in the disease course tends to yield better outcomes.

Exon-skipping drugs represent another standard therapy for DMD. These medications target the genetic mutations underlying the condition, aiming to promote the production of functional dystrophin proteins. However, this treatment approach is effective for only a subset of individuals with DMD.

Management of DMD may also involve addressing conditions such as sideways spinal curvature, known as scoliosis, which often arises as the disease progresses. Physicians monitor scoliosis using X-rays to determine the need for interventions such as braces or vests. In severe cases where spinal curvature impacts heart or breathing function, surgical intervention may be necessary.

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Progress and Support for Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the absence of a protein called dystrophin. It primarily affects boys, with symptom onset usually occurring between ages two and three. DMD leads to muscle weakness, making running, jumping, and walking challenging. Other symptoms include enlarged calves, a waddling gait, and inward spinal curvature.

Research has made strides since its discovery in the 1860s. Understanding the genetic cause paved the way for innovative approaches like CureDuchenne Link®, a data-integrated biobank aimed at accelerating research and improving care. Collecting biosamples and participant data provides a comprehensive resource for researchers working toward a cure. Participation is open to individuals living in the United States affected by DMD or Becker Muscular Dystrophy, regardless of mutation.

Every mutation matters in advancing research and developing effective therapies. CureDuchenne Link's community-centric approach and seamless participant experience contribute to a holistic understanding of the conditions, facilitating progress toward treatments and, ultimately, a cure.

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The Work of CureDuchenne

CureDuchenne is a nonprofit dedicated to saving the lives of boys with Duchenne muscular dystrophy (DMD). DMD belongs to a category of medical conditions called muscular dystrophies, a group of genetic disorders where muscles experience weakness. DMD complicates even the most basic activities. In addition, as the condition progresses, the muscles responsible for heart and breathing functions are also affected, further weakening the body.

This medical condition mostly affects boys. By age 12, most of them start to experience social isolation. Studies report that most boys with DMD do not make it to their late twenties, and those who do are usually confined to a wheelchair. There are about 20,000 boys living with DMD in the US, and more than 300,000 worldwide.

CureDuchenne is a renowned global innovation, research, and patient care organization committed to developing medical solutions that can improve and extend the lives of boys living with DMD. Together with its board of directors and scientific advisory board, the organization’s activities include extending patient ambulation in partnership with healthcare and medical professionals and pharmaceutical companies. Ambulation refers to the ability to walk without needing any assistance.

CureDuchenne’s diverse efforts include focusing on DMD treatments, investing in companies pursuing dystrophin-restoring and anti-inflammatory approaches. Dystrophin is the protein that stabilizes muscles.

The organization also seeks innovative technologies to overcome current therapy limitations. CureDuchenne Ventures’ investments have attracted over $3 billion in financing, leading to successful exits with venture capital, biotech, and pharmaceutical partners.

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What You Should Know about Podiatry

Podiatry, also called podiatric medicine and surgery, is a branch of medicine dealing with studying, diagnosing, and treating ankle and foot disorders. A specialist practicing podiatry is called a podiatrist. Notably, their official title is doctor of podiatric medicine (DPM). Additionally, they attend specialized schools and belong to distinct professional associations, not following the traditional medical school path.

Podiatrists treat people of varying ages for foot-related medical conditions. These include conditions such as arthritis, fractures and sprains, heel pains, and diabetes-related foot and leg nerve damage. Other conditions they treat include bunions, hammertoes, and Morton’s neuroma.

A bunion forms when the joint at the big toe’s base enlarges or dislocates, causing the toe to bend toward other toes. A hammertoe is an improperly bending toe. Morton’s neuroma is a pain or burning sensation arising from nerve issues between the third and fourth foot bones, common in runners. Tight shoes can also lead to this condition.

Aside from diagnosing and treating common foot and leg conditions, podiatrists can diagnose and treat foot and leg-related nail and skin diseases, deformities, tumors, and ulcers. Also, they develop flexible casts that provide steady support to the feet and legs for injuries like fractures and sprains.

Additionally, they discuss preventive foot care with patients. Moreover, if foot symptoms suggest disorders in other body systems, a podiatrist may facilitate referrals to other specialists.

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CureDuchenne Lauds FDA’s Approval of AGAMREE

CureDuchenne, a charitable organization that funds research efforts for the early diagnosis and treatment of Duchenne muscular dystrophy, lauded the US Food and Drug Administration for its AGAMREE (vamorolone) approval. CureDuchenne was involved during the early development stage of AGAMREE when it recognized that the drug has the potential to prevail over some challenges related to steroid treatment of Duchenne muscular dystrophy. To reduce side effects such as growth stunting and steroid-induced osteoporosis can have a significant impact on the quality of life of patients with Duchenne muscular dystrophy, a statement from CureDuchenne founder and CEO Debra Miller said.

AGAMREE is a corticosteroid that may be prescribed to patients at least two years old who have Duchenne muscular dystrophy. FDA’s approval of the drug was based on a series of studies conducted by ReveraGen Biopharma, which showed that young boys who were treated with vamorolone showed comparable efficacy with other steroids sold in the market. The study also revealed that vamorolone can help reduce the incidence of adverse events related to bone health.

AGAMREE will be available in the US, Canada, and Mexico by the first quarter of 2024.

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One GI Continues Strategic Push into Eastern US

Jeffrey Goffman has driven operations at Satellite Healthcare in San Jose, California, as chief executive office since 2021. When he is not supporting the Satellite Healthcare mission of improving the lives of those living with kidney disease, Jeffrey Goffman works as the vice chairman of One GI. The gastroenterology management services provider One GI announced a new partnership with Loudoun…

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Satellite Healthcare Opens Hawaiian High-Risk Nephrology Facility

As the CEO of Satellite Healthcare since 2021, Jeffrey Goffman strives to provide quality nephrology care, especially for high-risk patients. In 2022, Jeffrey Goffman opened Hawai‘i Kidney Care in Kuakini, Hawaii, to provide outpatient nephrology and dialysis services to residents. Hawai‘i Kidney Care fulfills multiple purposes, emphasizing nephrology care for patients with kidney disease and…

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Partnership Aims to Improve Kidney Care

In 2021, Jeffrey Goffman began his role as CEO of Satellite Healthcare in San Jose, California. One of Jeffrey Goffman’s achievements has been creating a partnership between the National Nephrology Alliance (NNA), a Satellite affiliate, and Nephrology Associates Medical Group (NAMG). The NNA, a managing organization, partnered with NAMG to expedite aspects of nephrology practices not involved…

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The National Nephrology Alliance

The CEO of Satellite Healthcare, Jeffrey Goffman, oversees programs and services to improve the quality of life and assist people with kidney disease. Jeffrey Goffman also led Satellite Healthcare in a landmark partnership with Nephrology Associates Medical Group (NAMG) to implement value-based care programs and optimize clinical and business goals through its satellite’s affiliate, the National…

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Travel Tips for People with Chronic Kidney Disease

A bachelor of science graduate in accounting from the University of Hartford, Jeffrey Goffman, has over 20 years of experience in comprehensive healthcare service provision. The CEO of Satellite Healthcare, Jeffrey Goffman, leverages his experience to offer premium services for people with kidney disease, including facilitating travel. Living with chronic kidney disease (CKD) should not deter…

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About In-Center Nocturnal Dialysis

The CEO of Satellite Healthcare, Jeffrey Goffman aims to provide top-quality care and patient satisfaction. The center offers training and support for treating and managing chronic kidney diseases in-center and at home. One of the services Jeffrey Goffman provides is in-center nocturnal dialysis. A standard hemodialysis treatment session takes three to four hours. In most cases, a patient is…

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Understanding the Role of Kidney Options Educators

With a bachelor’s degree in accounting from the University of Hartford, Jeffrey Goffman is the CEO of Satellite Healthcare in San Jose, California. One of the services offered at Jeffrey Goffman’s medical institution is kidney options educators. Kidney care educators are crucial in kidney disease management and treatment. The primary role of the educator is to provide education for the…

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Satellite Healthcare Introduces Multifaceted Kidney Dialysis Program

Satellite Healthcare Introduces Multifaceted Kidney Dialysis Program

The holder of a BS in accounting from the University of Hartford, Jeffrey Goffman is a California executive who has founded and led multiple healthcare ventures over the course of his career. Jeffrey Goffman serves as CEO of Satellite Healthcare, a nonprofit dedicated to bettering the lives of people with kidney diseases. As part of this care, Satellite Healthcare provides dialysis services. In…

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A Brief Overview of Hawai’i Kidney Care

A Brief Overview of Hawai’i Kidney Care

Healthcare executive Jeffrey Goffman has managed more than 150 outpatient and hospital-based healthcare provider facilities across the United States over the last two decades. Jeffrey Goffman serves as CEO of Satellite Healthcare, a non-profit organization based in San Jose, California, that provides kidney dialysis and allied healthcare services. Satellite Healthcare and three of its fellow…

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NNA-NAMG Partnership Promotes Value-Based Care in Nephrology Practice

Accomplished executive Jeffrey Goffman has over two decades of comprehensive experience in the healthcare industry. His years of work with private equity-backed companies gave him knowledge on creating and implementing strategies that create value for investors. Jeffrey Goffman leads Satellite Healthcare as CEO. Satellite Healthcare is a non-profit in nephrology practice, particularly in providing kidney dialysis services.

One of Satellite Healthcare’s affiliates, the National Nephrology Alliance (NNA), forged a strategic alliance with Nephrology Associates Medical Group (NAMG) in May 2022 to promote individualized patient-centered kidney care so that kidney patients will maintain healthy lives.

The collaboration leverages NNA’s unique business model to promote value-based patient care and innovative treatment approaches that deal with patients’ specific needs. The partnership aims to develop customized care at the earlier stage of their chronic kidney disease journeys, which will slow down their diseases’ progression, reduce the possibility of dialysis or hospitalization, improve their overall health, and cut their medical costs significantly.

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Satellite Healthcare Teams up with Nephrology Associates Medical Group

Jeffrey Goffman is a health care industry executive who has delivered solutions in areas such as physician practice management and health network efficiency. In 2021, Jeffrey Goffman assumed the role of CEO of Satellite Healthcare. The nonprofit company is a leader in the area of kidney dialysis therapy provided within home care settings.

In May 2022, Satellite Healthcare’s healthcare affiliate National Nephrology Alliance (NNA) announced that it was teaming up with Nephrology Associates Medical Group (NAMG) to advance care for patients with chronic kidney disease.

As a management services organization (MSO), NNA’s partnership with NAMG follows in the steps of affiliations with with other nephrology practices. These involve setting in place steps that boost practice growth, while ensuring a high standard of value-based care that incorporates customized patient treatment and advanced technologies. At the same time, the National Nephrology Alliance delivers administrative expertise that frees up resources and enables nephrologists to shift a focus from back office tasks to their highest priority, caring for patients.