Wasting Syndrome

The test will be used by providers and consumers to monitor muscle growth. BOCA RATON, Fla., March 13, 2024 /PRNewswire/ -- Physicians Lab, a provider of at-home lab testing for integrative medicine and wellness, launched a new at-home lab test that measures the analytes myostatin and follistatin. The test will show the current myostatin and follistatin levels and the ratio of myostatin to follistatin. The new test will be useful for monitoring muscle loss or growth in individuals. Continue Reading "Our research team has delivered another test that's convenient, reliable and has a fast turnaround time," Jaime Rios, CEO of Physicians Lab, said. "Our method is minimally invasive. We can provide valuable insight into a person's muscle development without burdening them by taking a large blood sample. Our team is excited to expand Physicians Lab solutions and to offer this at-home lab test to integrative providers and directly to health-conscious consumers." Physicians Lab provides convenient at-home lab testing that is precise, reliable and timely. Post this Myostatin, a member of the transforming growth factor-beta (TGF-β) superfamily, is a potent regulator of muscle growth. It acts primarily to inhibit muscle development, maintaining muscle size by inhibiting both the growth and differentiation of muscle cells. Assessing myostatin levels can provide valuable understanding of muscle homeostasis and the pathological mechanisms underlying muscle wasting diseases. Follistatin, serves as an antagonist to myostatin and other members of the TGF-β family. By binding to and inhibiting these growth factors, follistatin promotes muscle growth and has a critical role in muscle repair and regeneration. It's involved in a variety of biological processes beyond muscle growth, including reproduction and the regulation of inflammation. "GLP-1 has become a popular weight loss intervention," Rios said. "Understanding myostatin levels could be beneficial to patients on GLP-1 agonists like semaglutide, since up to 40 percent of the weight loss during therapy could come from lean mass loss, which includes muscle." Physicians Lab uses the technique called bloodspot microsampling. This method allows for the collection of blood samples in a minimally invasive manner. It requires only a small volume of blood that can be obtained from a simple finger prick. To determine the values of myostatin and follistatin, Physicians Lab uses a high-sensitivity Enzyme-Linked Immunosorbent Assay (ELISA) technique. This ensures accurate quantification of myostatin and follistatin levels in these small-volume samples. For more information about Physicians Lab and at-home lab testing solutions, visit . About Physicians Lab Physicians Lab, located in Boca Raton, FL, provides convenient at-home lab testing that is precise, reliable and timely. Physicians Lab serves physicians focused on integrative medicine and wellness for an aging population by shipping easy-to-use lab kits to patients' homes. Physicians Lab also delivers superior retail solutions direct to consumers who are actively seeking to manage their health and wellness. Contact: Jaime Rios, CEO (877) 326-5969 [email protected] SOURCE Physicians Lab

Pictured: A green, blue and pink ribbon representing rare disease advocacy/iStock, itakdalee Yesterday was Rare Disease Day 2024. It was a reminder of the importance of raising awareness for rare diseases—defined as those that affect fewer than 200,000 people in the U.S., but collectively affecting some 30 million Americans. While much has been achieved in improving access to lifesaving and life-changing treatments, much more remains to be done for patients with these disorders. Despite recent investment and breakthroughs for rare diseases, experts told BioSpace that helping a much larger patient population will require cross-sector support and desperately needed funding. We looked at some of the investments to date, as well as the biopharma industry’s promising pipelines and what is required to bring more groundbreaking therapies to patients with unmet medical needs. Not surprisingly, patient advocacy groups (PAGs) have been critical to the development of the first marketed drugs for certain rare diseases, including progeria and Friedreich’s ataxia. BioSpace spoke with leaders of PAGs formed by families coping with rare diseases, providing case studies on their partnerships with biopharma companies and the sharing of resources and expertise to maximize the impact of R&D and clinical trials. BioMarin Pharmaceutical picked the wrong week to disclose that it has been hit with a Department of Justice subpoena in connection with its sponsored testing programs for the rare disease therapies Vivizim—an FDA-approved enzyme replacement therapy for the treatment of Morquio A syndrome—and Naglazyme, an enzyme replacement therapy approved for mucopolysaccharidosis VI. Vivizim was approved in 2014 and Naglazyme was approved in 2005. In one of 2023’s most highly anticipated FDA approvals, Sarepta Therapeutics’ Elevidys got approved in June as the first gene therapy for DMD targeting the underlying cause of the progressive genetic muscle-wasting disease, despite failing to meet the primary functional endpoint in a randomized trial. With a $3.2 million per patient price tag, Elevidys—developed and commercialized in partnership with Roche—also has the dubious distinction of being one of the world’s most expensive medicines. Sarepta CEO Doug Ingram told investors in Wednesday’s fourth quarter and full-year 2023 earnings call that the launch of Elevidys was “particularly impressive,” bringing in over $200 million in revenue since mid-2023, especially given that its label is “limited to four- and five-year-olds, representing only about 3% or so of the total Duchenne population.” The successful launch of Elevidys “shows that gene therapy can be commercially viable, providing hope for those patients with Duchenne, and for all those with genetic conditions with unmet needs,” Sarepta Chief Customer Officer Dallan Murray said during the call. However, Murray cautioned analysts to “not use this current younger population as a frame of reference for our market potential in the overall population.” At the end of last year, Sarepta submitted a supplemental BLA seeking to expand the gene therapy’s label by removing age and ambulation restrictions, while transitioning Elevidys from an accelerated to a traditional approval. The FDA has set a PDUFA date of June 21, 2024, and will not convene an advisory committee meeting to discuss the efficacy supplement to the BLA. “Hopefully, by July, we will have a broader label and we will be able to serve a much greater percentage of the [DMD] population,” Ingram said. Next, Sarepta should work on lowering the hefty price of Elevidys and ensuring equitable access to as many patients as possible who will benefit from the therapy. Greg Slabodkin is the News Editor at BioSpace. You can reach him at greg.slabodkin@biospace.com. Follow him on LinkedIn.

– Patient dosing expected to begin mid-to-late Q1 2024 – CHARLESTOWN, Mass., Jan. 16, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it has been granted orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate. “Obtaining ODD status for SGT-003, along with Fast Track Designation granted last month, furthers our efforts to meet the ongoing challenge of treating this devastating disease as expeditiously as possible,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences. “These designations are important milestones for Solid, supporting the continued development of next-generation therapies for Duchenne.” The company is currently in the process of securing approvals from the institutional review boards (IRB) at the clinical trial sites for the planned Phase 1/2 clinical trial of SGT-003 and expects to commence patient screening shortly thereafter. Patient dosing in the trial is expected to commence in mid-to-late first quarter of 2024. “SGT-003 therapy stands out among other Duchenne gene therapy candidates by leveraging a novel capsid and a muscle tropic vector delivering a microdystrophin that incorporates a neuronal Nitric Oxide Synthase (nNOS) binding domain. These attributes among others, have the potential to yield both more potent transduction than historical approaches, and a microdystrophin that may be able to more fully address muscle resiliency,” said Gabriel Brooks, M.D., Chief Medical Officer at Solid Biosciences. “Obtaining ODD status is an exciting development that we believe will aid our efforts to bring advanced treatment options to those patients affected by Duchenne.” About SGT-003SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding a shortened form of the dystrophin protein (microdystrophin), containing the R16-R17 nNOS binding domain. Preclinical data suggests this may be important for both muscular function and durability of benefit in patients. About Orphan Drug DesignationThe FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended to treat a rare disease or condition that affects fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including specified financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval. About DMDDuchenne is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. Duchenne is a progressive, irreversible, and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 5,000 to 15,000 cases in the United States alone.About Solid BiosciencesSolid Biosciences is a life sciences company focused on advancing a portfolio of gene therapy candidates and neuromuscular and cardiac programs, including SGT-003, for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, AVB-202-TT for the treatment of Friedreich’s ataxia, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com. Forward-Looking StatementsThis press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the company; the ability to successfully achieve and execute on the company’s priorities and achieve key clinical milestones; the company’s SGT-003 program, including securing IRB approval at clinical sites, initiating patient screening and initiating dosing, and the potential benefits of SGT-003; the potential benefits of ODD status; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the ability to recognize the anticipated benefits of Solid’s acquisition of AavantiBio; the company’s ability to advance SGT-003, SGT-501, AVB-401, AVB-202-TT and other preclinical programs and capsid libraries on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies of the company’s product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, SGT-501, AVB-401, AVB-202-TT and other candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof and should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. Solid Biosciences Contact:Leah MonteiroVP, Investor Relations and Communications617-766-3430lmonteiro@solidbio.com