Endocrinology Center of Excellence

Supporting patients living with a rare endocrinology condition

Orsini’s Endocrinology Center of Excellence launched in 2015 with our first pediatric growth hormone program. Today we support multiple endocrinology programs across several rare conditions. Our dedicated Therapy Care Teams work with the physician’s office and insurance on a patient’s behalf so that our patients and their family have more time to focus on their treatment and quality of life. We are here to listen, provide counseling on treatment, including side-effect management, and assist with sourcing third-party financial assistance.

Explore Our Endocrinology Therapies

Benefits

Improving the Patient Experience, Every Time

Dedicated Care Team

Receive personalized support from a dedicated Therapy Care Team, including a primary pharmacy contact, clinical education, side-effects management, and scheduled refill calls.

Home Health Nursing

Access nationwide nursing support for in-home treatment or self-administration training. Our nurses provide clinical support to help patients achieve their treatment goals.

Insurance Coverage

Secure treatment coverage with assistance navigating payor and plan requirements. Our team proactively works with prescribers to obtain prior authorization and support appeals.

Free Care Overnight Shipping

Ensure medication and essential medical supplies arrive when and where patients need them through coordination across prescribers, patients, and care teams to arrange free overnight shipping.

Financial Assistance

Minimize financial exposure through financial assistance programs. We successfully identify and assist in enrolling patients into manufacturer co-pay and foundation support programs.

24/7/365 Access to a Pharmacist

Gain continuous access to a pharmacist, 24/7/365, ensuring ongoing care and clinical support for patients and prescribers to achieve better outcomes.

Support

Endocrinology Conditions Supported

Explore the rare endocrinology conditions and therapies Orsini supports below.

About Acromegaly

Acromegaly develops when a benign pituitary tumor forms and secretes increased levels of growth hormone (GH). Acromegaly is characterized by somatic overgrowth (often in the hands, feet and face), and other physical changes, joint pain, metabolic disorders, cardiovascular disease, and respiratory problems, among others. When left untreated, acromegaly can lead to premature mortality. 

Acromegaly Therapies Supported

Full Name	PALSONIFY™ (paltusotine)
Drug	paltusotine
Manufacturer	Crinetics Pharmaceuticals, Inc.
Route of Administration	Oral
Site of Care	Home
Approved Indication	The treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option
Disease	Acromegaly
Therapeutic Area	Endocrinology
Enrollment Form Link	Enrollment Form
Phone Number	847-239-5680
Fax Number	847-892-1203
Product Website	palsonify.com

About Cushing’s Syndrome

Cushing’s syndrome, also known as hypercortisolism, is a rare condition that occurs when the body produces too much of the hormone cortisol. This can lead to bone loss, weight gain, high blood pressure, high blood sugar, and increased thirst, among other symptoms.

Cushing’s Syndrome Therapies Supported

Full Name	Mifepristone
Drug	Mifepristone
Manufacturer	Generic
Route of Administration	Oral
Site of Care	Home
Approved Indication	Control hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing’s syndrome who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery
Disease	Cushing's Syndrome
Therapeutic Area	Endocrinology, Metabolic Disorder
Enrollment Form Link	Mifepristone Enrollment Form
Phone Number	(888) 204-7730
Fax Number	(877) 218-6288

About Hypoparathyroidism

Hypoparathyroidism is a rare endocrine disorder characterized by insufficient production or action of parathyroid hormone (PTH), a key regulator of calcium, phosphorus, and vitamin D metabolism. The condition leads to low calcium levels and elevated phosphorus levels in the blood and causes muscle aches and spasms. It may also cause kidney complications, cognitive impairment, and cardiac arrhythmias.

Hypoparathyroidism Therapies Supported

Full Name	YORVIPATH® (palopegteriparatide)
Drug	Yorvipath
Manufacturer	Ascendis Pharma
Route of Administration	Subcutaneous
Site of Care	Home
Approved Indication	The treatment of hypoparathyroidism in adults
Disease	Hypoparathyroidism
Therapeutic Area	Endocrinology
Enrollment Form Link	Enrollment Form
Phone Number	888-204-7802
Fax Number	877-471-8175
Product Website	yorvipathhcp.com
Patient Resources	HypoPARAthyroidism Association

About Phenylketonuria (PKU)

PKU is a rare genetic disorder characterized by an inability to metabolize the amino acid phenylalanine (Phe), which causes Phe to accumulate in the body. As a result, those living with PKU can experience a wide variety of symptoms if left untreated, including severe brain damage and intellectual disabilities.

PKU Therapies Supported

Sapropterin Featured Image_Orsini Website

Full Name	Sapropterin
Drug	sapropterin dihydrochloride
Manufacturer	Generic
Route of Administration	Oral
Site of Care	Home
Approved Indication	Reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU), in conjunction with a Phe-restricted diet
Disease	Phenylketonuria (PKU)
Therapeutic Area	Endocrinology, Metabolic Disorder
Enrollment Form Link	Enrollment Form
Phone Number	(773) 598-6135
Fax Number	(877) 299-8203
Patient Resources	Alliance of PKU Families, flok, and National PKU Alliance

Sephience Featured Image 2_Orsini Website

Full Name	SEPHIENCE™ (sepiapterin)
Drug	sepiapterin
Manufacturer	PTC Therapeutics
Route of Administration	Oral
Site of Care	Home
Approved Indication	Treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU)
Disease	Phenylketonuria (PKU)
Therapeutic Area	Endocrinology, Metabolic Disorder
Enrollment Form Link	Enrollment Form
Phone Number	(847) 521-4499
Fax Number	(888) 726-9306
Product Website	sephience.com
Patient Resources	Alliance of PKU Families, flok, and National PKU Alliance

About Tumor-induced Osteomalacia (TIO)

Tumor-induced osteomalacia is a rare paraneoplastic syndrome characterized by weakened bones due to phosphate wasting. Tumors produce a hormone-like substance causing renal phosphate loss, leading to bone softening, fractures, and muscle weakness. Diagnosis involves identifying the tumor through imaging and blood tests.

TIO Therapies Supported

Full Name	Crysvita® (burosumab-twza)
Drug	Crysvita
Manufacturer	Kyowa Kirin North America
Route of Administration	Subcutaneous
Site of Care	Home or Healthcare Facility
Approved Indication	The treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older and FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adult and pediatric patients 2 years of age and older
Disease	Tumor-induced Osteomalacia (TIO); X-linked Hypophosphatemia (XLH)
Therapeutic Area	Endocrinology & Bone Specialty
Enrollment Form Link	Enrollment Form
Phone Number	800-240-7614
Fax Number	877-793-4897
Product Website	crysvitahcp.com

About Type 1 Diabetes (T1D)

Type 1 diabetes is a chronic condition where the immune system mistakenly attacks and destroys insulin-producing cells in the pancreas. This results in insufficient insulin, leading to high blood sugar levels. Symptoms include increased thirst, frequent urination, and weight loss.

T1D Therapies Supported

Full Name	Tzield® (teplizumab-mzwv)
Drug	Tzield
Manufacturer	Sanofi
Route of Administration	Intravenous
Site of Care	Home or Healthcare Facility
Approved Indication	Delay the onset of Stage 3 type 1 diabetes (T1D) in adults and pediatric patients aged 8 years and older with Stage 2 T1D
Disease	Type 1 Diabetes (T1D)
Therapeutic Area	Endocrinology & Bone Specialty
Enrollment Form Link	Tzield Enrollment Form
Phone Number	800-670-5321
Fax Number	877-655-4364
Product Website	tzield.com

About X-linked Hypophosphatemia (XLH)

X-linked hypophosphatemia is a genetic disorder causing low levels of phosphate in the blood. It results from mutations in the PHEX gene, affecting phosphate reabsorption in the kidneys. XLH leads to weakened bones, bowed legs, and dental issues.

XLH Therapies Supported

Full Name	Crysvita® (burosumab-twza)
Drug	Crysvita
Manufacturer	Kyowa Kirin North America
Route of Administration	Subcutaneous
Site of Care	Home or Healthcare Facility
Approved Indication	The treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older and FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adult and pediatric patients 2 years of age and older
Disease	Tumor-induced Osteomalacia (TIO); X-linked Hypophosphatemia (XLH)
Therapeutic Area	Endocrinology & Bone Specialty
Enrollment Form Link	Enrollment Form
Phone Number	800-240-7614
Fax Number	877-793-4897
Product Website	crysvitahcp.com

Patient Advocacy Partners

Acromegaly Community is on a mission to provide an emotional and communal support network for people touched by Acromegaly. They offer a central location for medical information on issues including: surgery, medication, radiation, and post diagnosis support. Most importantly, they work to provide a network of emotional support for Acromegaly patients, their friends and their family.

Learn more at acromegalycommunity.org.

The Amyloidosis Research improves the lives of patients with amyloidosis and accelerates cutting edge research through collaborative, groundbreaking initiatives. ARC provides support, information, and resources to patients and caregivers, empowering them to access the best care possible.

Learn more at aci.org

Cure SMA is dedicated to finding treatments and a cure for spinal muscular atrophy (SMA) by funding research and supporting families. Since 1984, Cure SMA has grown to be the largest U.S.-based network of individuals, families, clinicians, and research scientists working together to advance SMA research, support individuals and families impacted by SMA, and educate public and professional communities about SMA.

Learn more at www.curesma.org

The US Hereditary Angioedema Association (HAEA) is a 501 (c)(3) non-profit advocacy and research organization serving people living with the rare genetic condition, Hereditary Angioedema (HAE), and their families. Founded and staffed by people with HAE and caregivers, the US HAEA is committed to actively engaging our community in a wide variety of grassroots activities that promote HAE education and awareness.

Learn more at haea.org

The HypoPARAthyroidism Association is a non-profit patient association working to improve the lives of people impacted by hypoPARAthyroidism through education, support, research, and advocacy. They raise awareness of hypoPARAthyroidism and bring change to the lives of those affected.

Learn more at hypopara.org

Jett Foundation empowers people and families impacted by Duchenne muscular dystrophy through the development of transformative programming, educational opportunities, and ongoing support for every stage of a Duchenne journey.

Learn more at jettfoundation.org

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions. For 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease.

Learn more at mda.org

The National MPS Society is committed to making a difference in the lives of MPS families through support, research, education, and advocacy. Families from around the world gain a better understanding of these rare genetically determined diseases through the Society’s assistance in linking them with healthcare professionals, researchers, and, perhaps most importantly, each other.

Learn more at mpssociety.org

The National Niemann-Pick Disease Foundation, Inc. (NNPDF) is a non-profit, patient advocacy and family support organization dedicated to supporting and empowering patients and families affected by Niemann-Pick disease, through education, collaboration and research. Founded in 1993, NNPDF serves families throughout the nation at all stages of their Niemann-Pick journey.

Learn more at nnpdf.org

The National PKU Alliance (NPKUA) works to improves the lives of individuals with PKU, pursue a cure by expanding research, and provide education and support to individuals living with PKU and their caregivers. Since its inception in 2008, NPKUA has supported numerous successful fundraising events, conferences, and internal support programs and has remained steadfast in its support of research endeavors.

Learn more at npkua.org

Parent Project Muscular Dystrophy (PPMD) is the largest nonprofit organization in the United States focused entirely on Duchenne muscular dystrophy. They take a comprehensive approach in the fight against Duchenne—funding research, raising awareness, promoting advocacy, connecting the community, and broadening treatment options.

Learn more at parentprojectmd.org

The Pompe Alliance is a Public Services Organization dedicated to the ideas of support, empowerment, and progress. It seeks to provide supportive services and information to patients, caregivers, medical professionals, and community stakeholders through a number of initiatives: financial assistance, advocacy, education, mentoring, and research recruitment

Learn more at pompealliance.com

The mission of the Pulmonary Fibrosis Foundation is to accelerate the development of new treatments and ultimately a cure for pulmonary fibrosis. Until this goal is achieved, the PFF is committed to advancing improved care of patients with PF and providing unequaled support and education resources for patients, caregivers, family members, and health care providers.

Learn more at pulmonaryfibrosis.org

The United Pompe Foundation was formed to assist patients and/or their families with medical costs and other expenses that these patents and families face and may not be able to cover, or fully cover, through their insurance.

Learn more at unitedpompe.com