Latest news with #PPMD
Yahoo
4 hours ago
- Business
- Yahoo
UPSHER-SMITH SHOWCASES ITS COMMITMENT TO DUCHENNE MUSCULAR DYSTROPHY COMMUNITY AS A TRUSTED PARTNER IN PEDIATRIC RARE DISEASES AT THE PARENT PROJECT MUSCULAR DYSTROPHY (PPMD) 2025 ANNUAL CONFERENCE
Visit Upsher-Smith at the PPMD 2025 Annual Conference Resource Fair to Learn More About Support Services and Resources Offered for Deflazacort Tablets MAPLE GROVE, Minn., June 16, 2025 /PRNewswire/ -- Upsher-Smith Laboratories, LLC (Upsher-Smith) today announced its participation at the Parent Project Muscular Dystrophy (PPMD) 2025 Annual Conference from June 19-21, 2025 in Las Vegas, Nevada. Representatives from Upsher-Smith will be available to share information about Deflazacort Tablets and discuss the Company's intent and commitment to bring its best-in-class rare disease support and services to the Duchenne muscular dystrophy community. "Experience matters in rare disease. Founded in 1919, Upsher-Smith has been serving patients for more than 100 years and has a long-standing commitment to supporting the unique needs of rare disease communities," said Jim Maahs, Head of Commercial, Upsher-Smith. "As a trusted partner in pediatric rare disease, we invite attendees to visit us during this year's PPMD Annual Conference to learn how Upsher-Smith is committing to elevating the standard of support for patients and families affected by Duchenne muscular dystrophy (DMD) by offering high-quality generic treatment options that match or exceed the level of support services provided by innovator brands." About Upsher-Smith's Deflazacort Tablets Deflazacort Tablets from Upsher-Smith are an AB-rated generic version of Emflaza® (deflazacort) Tablets.1 This means they have the same active ingredient in the same strength and dosage form and provide the same efficacy, tolerability, and safety as Emflaza®.1 Deflazacort Tablets are indicated for the treatment of DMD in patients 5 years of age and older. Upsher-Smith's Deflazacort Tablets are available in 6 mg, 18 mg, 30 mg and 36 mg strengths. Deflazacort tablets are manufactured by Upsher-Smith in Minnesota. INDICATION & IMPORTANT SAFETY INFORMATION FOR DEFLAZACORT TABLETS INDICATION DEFLAZACORT TABLETS are a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older. IMPORTANT SAFETY INFORMATION Contraindications: DEFLAZACORT TABLETS are contraindicated in patients with a hypersensitivity to deflazacort or any of the inactive ingredients. Warnings & Precautions Alterations in Endocrine Function: Corticosteroids, such as DEFLAZACORT, can cause serious and life-threatening alterations in endocrine function, especially with chronic use. Monitor patients receiving DEFLAZACORT for Cushing's syndrome, hyperglycemia, and adrenal insufficiency after DEFLAZACORT withdrawal. In addition, patients with hypopituitarism, primary adrenal insufficiency or congenital adrenal hyperplasia, altered thyroid function, or pheochromocytoma may be at increased risk for adverse endocrine events. Acute adrenal insufficiency or "withdrawal syndrome" can occur if corticosteroids are withdrawn abruptly and can be fatal. The risk is reduced by gradually tapering the corticosteroid dose when withdrawing treatment. During times of medical stress, corticosteroid dosage may need to be increased. Immunosuppression and Increased Risk of Infection: Increased risk of new, exacerbation, dissemination, or reactivation of latent infections, which can be severe and at times fatal; signs and symptoms of infection may be masked. Monitor for the development of infection and consider DEFLAZACORT withdrawal or dosage reduction as needed. Tell patients and/or caregivers to inform their healthcare provider if the patient has had recent or ongoing infections or if they have recently received a vaccine. Warn patients who are on corticosteroids who have not had chickenpox or measles to avoid exposure to chickenpox or measles and to alert their healthcare provider immediately if they are exposed. Alterations in Cardiovascular/Renal Function: Monitor for elevated blood pressure. Dietary salt restriction and potassium supplementation may be necessary. DEFLAZACORT should be used with caution in patients with congestive heart failure, hypertension, or renal insufficiency. Gastrointestinal Perforation: Increased risk of gastrointestinal perforation during corticosteroid use in patients with certain gastrointestinal disorders such as active or latent peptic ulcers, diverticulitis, recent intestinal anastomoses, and inflammatory bowel disease. Signs and symptoms may be masked. Behavioral and Mood Disturbances: May include euphoria, insomnia, mood swings, personality changes, severe depression, and psychosis. Symptoms typically emerge within a few days or weeks of starting treatment and may be dose-related. Inform patients or caregivers of the potential for behavioral and mood changes. Effects on Bones: The risk of osteoporosis increases with prolonged use of DEFLAZACORT, which can predispose patients to vertebral and long bone fractures. Monitor for decreases in bone density with chronic use of DEFLAZACORT. Encourage patients to seek medical attention if symptoms develop or worsen. Ophthalmic Effects: May include cataract formation, ocular infections, and glaucoma. If treatment with corticosteroids, including DEFLAZACORT, are continued for more than 6 weeks, monitor intraocular pressure. Vaccination: Do not administer live or live attenuated vaccines to patients receiving immunosuppressive doses of corticosteroids. Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting DEFLAZACORT. Serious Skin Rashes: Toxic epidermal necrolysis has been reported with the use of deflazacort. Discontinue at the first sign of rash, unless the rash is clearly not drug related. Effects on Growth and Development: Long-term use of corticosteroids, including DEFLAZACORT, can have negative effects on growth and development in children. Thromboembolic Events: Observational studies have shown an increased risk of thromboembolism. Use DEFLAZACORT with caution in patients who have or may be predisposed to thromboembolic disorders. Adverse Reactions: The most common adverse reactions (≥10% for DEFLAZACORT and greater than placebo) are Cushingoid appearance, weight increased, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, central obesity, and nasopharyngitis. Drug Interactions: Give one third of the recommended dose of DEFLAZACORT when DEFLAZACORT is administered with strong or moderate CYP3A4 inhibitors. Avoid use of strong or moderate CYP3A4 inducers with DEFLAZACORT, as they may reduce efficacy. This safety information is not comprehensive. Please see the full Prescribing Information for DEFLAZACORT TABLETS. You can also visit or call 1-888-650-3789. You are encouraged to report suspected adverse reactions to Upsher-Smith Laboratories, LLC at 1-888-650-3789 or to the FDA by visiting About Upsher-SmithUpsher-Smith Laboratories, LLC, now a member of Bora Group, is a trusted U.S. pharmaceutical company that strives to improve the health and lives of patients through an unwavering commitment to high-quality products and sustainable growth. We bring generics and brands to a wide array of customers, always backed by our attentive level of service, strong industry relationships, and dedication to uninterrupted supply. For more information, visit About Bora Founded in 2007, Bora Pharmaceutical Co., Ltd. ("Bora" or "the Company", now is the largest pharmaceutical manufacturer in Taiwan with well-connected global distribution to supply more than 100 countries around the world. Bora is dedicated to becoming a global leader in pharmaceutical manufacturing by offering its clients the best quality, efficiency and reliability. For more information, visit Reference: 1. Approved Drug Products with Therapeutic Equivalence Evaluations | Orange Book Food and Drug Administration Website. gov/scripts/cder/ob/ Emflaza is a registered trademark of PTC Therapeutics, Inc. View original content to download multimedia: SOURCE Upsher-Smith Laboratories, LLC
National Post
13 hours ago
- Business
- National Post
Satellos Bioscience to Participate in PPMD's Annual Conference in Las Vegas
Article content TORONTO — Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (' Satellos ' or the ' Company '), a biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, today announced its participation in the 2025 Parent Project Muscular Dystrophy (PPMD) Annual Conference, taking place June 19–21 in Las Vegas, Nevada. Article content The PPMD Annual Conference brings together families, researchers, clinicians, and companies committed to improving outcomes for people living with Duchenne. Satellos will be onsite to engage with the Duchenne community, listen to families' experiences, and share updates on its science and mission to regenerate muscle from within. Article content Article content 'We're looking forward to connecting in person with families and advocates who are at the heart of this work,' said Frank Gleeson, Co-founder and CEO of Satellos. 'This conference is a powerful reminder of why we're focused on developing a new approach to treat Duchenne — one that aims to restore the body's ability to repair muscle.' Article content Attendees can meet members of the Satellos team throughout the event and learn more by visiting the company's booth in the exhibition hall. Article content About Satellos Bioscience Inc. Article content Satellos is a clinical-stage drug development company focused on restoring natural muscle repair and regeneration in degenerative muscle diseases. Through its research, Satellos has developed SAT-3247, a first-of-its-kind, orally administered small molecule drug designed to address deficits in muscle repair and regeneration. SAT-3247 targets AAK1, a key protein that Satellos has identified as capable of replacing the signal normally provided by dystrophin in muscle stem cells to effect repair and regeneration. By restoring this missing dystrophin signal in DMD, SAT-3247 enables muscle stem cells to divide properly and more efficiently, promoting natural muscle repair and regeneration. SAT-3247 is currently in clinical development as a potential disease-modifying treatment initially for DMD. Satellos also is leveraging its proprietary discovery platform MyoReGenX™ to identify additional muscle diseases or injury conditions where restoring muscle repair and regeneration may have therapeutic benefit and represent future clinical development opportunities. For more information, visit Article content This press release includes forward-looking information or forward-looking statements within the meaning of applicable securities laws regarding Satellos and its business, which may include, but are not limited to, statements regarding the potential for SAT-3247 to represent a disease modifying approach to the therapeutic treatment of people living with Duchenne; anticipated benefits to patients from a small molecule treatment for Duchenne; the advancement SAT-3247 through clinical trials; the pharmacodynamic properties and mechanism-of-action of SAT-3247; the potential of our approach in other degenerative muscle diseases; its/their prospective impact on Duchenne patients, patients with other degenerative muscle disease or muscle injury or trauma, and on muscle regeneration generally; and Satellos' technologies and drug development plans. All statements that are, or information which is, not historical facts, including without limitation, statements regarding future estimates, plans, programs, forecasts, projections, objectives, assumptions, expectations or beliefs of future performance, occurrences or developments, are 'forward-looking information or statements.' Often but not always, forward-looking information or statements can be identified by the use of words such as 'shall', 'intends', 'believe', 'plan', 'expect', 'intend', 'estimate', 'anticipate', 'potential', 'prospective' , 'assert' or any variations (including negative or plural variations) of such words and phrases, or state that certain actions, events or results 'may', 'might', 'can', 'could', 'would' or 'will' be taken, occur, lead to, result in, or, be achieved. Such statements are based on the current expectations and views of future events of the management of the Company. They are based on assumptions and subject to risks and uncertainties. Although management believes that the assumptions underlying these statements are reasonable, they may prove to be incorrect. The forward-looking events and circumstances discussed in this release, may not occur and could differ materially as a result of known and unknown risk factors and uncertainties affecting the Company, including, without limitation, risks relating to the pharmaceutical and bioscience industry (including the risks associated with preclinical and clinical trials and regulatory approvals), and the research and development of therapeutics, the results of preclinical and clinical trials, general market conditions and equity markets, economic factors and management's ability to manage and to operate the business of the Company generally, including inflation and the costs of operating a biopharma business, and those risks listed in the 'Risk Factors' section of Satellos' Annual Information Form dated March 26, 2025 (which is located on Satellos' profile at Although Satellos has attempted to identify important factors that could cause actual actions, events or results to differ materially from those described in forward-looking statements, there may be other factors that cause actions, events or results to differ from those anticipated, estimated or intended. Accordingly, readers should not place undue reliance on any forward-looking statements or information. No forward-looking statement can be guaranteed. Except as required by applicable securities laws, forward-looking statements speak only as of the date on which they are made and Satellos does not undertake any obligation to publicly update or revise any forward-looking statement, whether resulting from new information, future events, or otherwise. Article content Article content Article content Article content Article content Contacts Article content Investors: Article content Liz Williams, CFO, Article content ir@ Article content Article content Media: Article content Emily Williams, Senior Director, Communications, Article content Article content Article content
Associated Press
12-05-2025
- Health
- Associated Press
Parent Project Muscular Dystrophy Announces Publication of Updated PJ Nicholoff Steroid Protocol, Advancing Care for Individuals with Duchenne
WASHINGTON, May 12, 2025 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), in partnership with the OPTIMIZE DMD Consortium, an international consortium that aims to advance the standard of care, education, advocacy and research in endocrine and bone health for individuals with Duchenne, is proud to announce the publication of the updated version of the PJ Nicholoff Steroid Protocol for Healthcare Providers. This resource has been a lifeline for families, providing doctors with clear guidance on managing steroid dosing during illness and emergencies, and is designed to enhance the clinical management of adrenal insufficiency and steroid stress dosing for individuals on long-term corticosteroid therapies. This milestone comes as PPMD celebrates the 10th anniversary of the first publication of the PJ Nicholoff Steroid Protocol. The PJ Nicholoff Steroid Protocol has been instrumental in educating medical professionals on the unique care considerations for patients with Duchenne on long-term steroids who may have accompanying adrenal suppression. The newly revised edition reflects the latest advancements in clinical research, changes in the approved therapy landscape, and critical safety measures to improve patient outcomes. PPMD is grateful to those working to keep this information up to date and relative to our Duchenne community. 'At PPMD, we are committed to ensuring that every individual with Duchenne receives the highest standard of care,' said Rachel Schrader, MS, APRN, CPNP-PC, PPMD's Vice President, Clinical Care & Education. 'This significant update to the PJ Nicholoff Steroid Protocol reflects the rapidly changing landscape of approved therapies and subsequent changes to clinical care. The Protocol provides expert recommendations to better equip healthcare professionals and caregivers with the most up-to-date knowledge to safely manage patients experiencing illness, injury, or safely navigating other substantive changes to their steroid regimen.' The revised the PJ Nicholoff Steroid Protocol expands beyond the previous iteration and includes six 'Critical Concepts,' including: 'The PJ Nicholoff Protocol is a 'how-to' guide designed to assist healthcare professionals in the prevention of adrenal insufficiency for people with Duchenne, a potentially serious complication of corticosteroid therapy,' said Leanne Ward, MD, FRCPC, a pediatric endocrinologist at the Children's Hospital of Eastern Ontario and Founder and Chair of the OPTIMIZE DMD Consortium. 'The 2025 edition, developed under the expert leadership of Dr. David Weber (Children's Hospital of Philadelphia) and Dr. Anne Marie Sbrocchi (Montreal Children's Hospital), provides important updates including the management of adrenal insufficiency for individuals receiving the novel steroid, vamorolone. On behalf of the OPTIMIZE DMD Consortium, we are so grateful to the Nicholoff family for their enduring support towards international efforts aimed at the prevention of adrenal insufficiency in Duchenne muscular dystrophy.' The updated healthcare provider-facing protocol is available for download on PPMD's website here. PPMD will be hosting a webinar on May 30, 2025, at 1:00 PM ET with leading experts to discuss its implementation in clinical practice. Register for the webinar here. PPMD encourages families, caregivers, and healthcare providers to access this vital resource to ensure the safest care for individuals with Duchenne. For more information or to access the PJ Nicholoff Steroid Protocol, visit About Parent Project Muscular Dystrophy Duchenne is a genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne. We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won eight FDA approvals. Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at Follow PPMD on Facebook, Twitter, Instagram, and YouTube. View original content to download multimedia: SOURCE Parent Project Muscular Dystrophy (PPMD)
