Tabes Dorsalis

Researchers have developed a novel screening method to identify a drug that mimics the effects of exercise on muscle and bone. Locamidazole (LAMZ) improved muscle width and function and promoted bone formation while reducing bone resorption. LAMZ serves as a potential therapy for the treatment of locomotor diseases, such as sarcopenia and osteoporosis. Maintaining a regular workout routine can help you look and feel great -- but did you know that exercise also helps maintain your muscles and bones? People who are unable to engage in physical activity experience weakening of the muscles and bones, a condition known as locomotor frailty. Recently, researchers in Japan have identified a new drug that may aid in the treatment of locomotor frailty by inducing similar effects as exercise. Physical inactivity can result in a weakening of the muscles (known as sarcopenia) and bones (known as osteoporosis). Exercise dispels this frailty, increasing muscle strength and promoting bone formation while suppressing bone resorption. However, exercise therapy cannot be applied to all clinical cases. Drug therapy may be helpful in treating sarcopenia and osteoporosis, especially when patients have cerebrovascular disease, dementia, or when they have already become bedridden. However, there is no single drug that addresses both tissues simultaneously. In a new study published in Bone Research, researchers from Tokyo Medical and Dental University(TMDU) developed a novel drug screening system to identify a compound that mimics the changes in muscle and bone that occur as a result of exercise. Using the screening system, the researchers identified the aminoindazole derivative locamidazole (LAMZ). LAMZ was capable of stimulating the growth of muscle cells and bone-forming cells, osteoblasts, while suppressing the growth of bone-resorbing cells, osteoclasts. When LAMZ was administrated to mice orally, it was successfully transmitted into the blood, with no obvious side effects. "We were pleased to find that LAMZ-treated mice exhibited larger muscle fiber width, greater maximal muscle strength, a higher rate of bone formation, and lower bone resorption activity," says lead author of the study Takehito Ono. The research team further addressed the mode of function of LAMZ and found that LAMZ mimics calcium and PGC-1α signaling pathways. These pathways are activated during exercise and stimulate expression of downstream molecules that are involved in the maintenance of muscle and bone. To investigate whether LAMZ can treat locomotor frailty, LAMZ was administrated to an animal model of sarcopenia and osteoporosis. "Both oral and subcutaneous administration of the drug improved the muscle and bone of mice with locomotor frailty," says senior author Tomoki Nakashima. Taken together, the research team's findings show that LAMZ represents a potential therapeutic method for the treatment of locomotor frailty by mimicking exercise.

While Sarep­ta has three an­ti­sense oligonu­cleotide ther­a­pies ap­proved — al­beit not with­out con­tro­ver­sy — for Duchenne mus­cu­lar dy­s­tro­phy, it has been work­ing on a long-term treat­ment in the form of a gene ther­a­py. Af­ter post­ing pos­i­tive re­sults for its Roche-part­nered gene ther­a­py, dubbed SRP-9001, ear­li­er this month, the Cam­bridge, MA-based biotech says it now in­tends to pur­sue an ac­cel­er­at­ed ap­proval for its lat­est Duchenne treat­ment. Sarep­ta shares $SRPT rose about 10% on the news in ear­ly Fri­day trad­ing. Baird an­a­lyst Bri­an Sko­r­ney wrote in a note that he be­lieved Sarep­ta is “well-aligned with the FDA on the plan to file this fall, with the po­ten­tial for an ap­proval de­ci­sion by mid-2023.” Sko­r­ney added: Giv­en this pos­i­tive com­men­tary, we think that once the BLA is sub­mit­ted, around Oc­to­ber at the lat­est, FDA ac­cep­tance for re­view will be a near cer­tain­ty. Al­though man­age­ment not­ed they have not dis­cussed the pos­si­bil­i­ty of an Ad­vi­so­ry Com­mit­tee Meet­ing with the agency, they feel that an Ad­Com will be a near cer­tain­ty giv­en the grav­i­ty of the re­view, and we con­cur with this ex­pec­ta­tion. Sko­r­ney al­so not­ed that the fil­ing may in­clude all am­bu­lant Duchenne pa­tients sans age re­stric­tions, though its cur­rent tri­als are in pa­tients ages 4-7. In ear­ly 2021, SRP-9001 failed in an ini­tial Phase II study, which Sarep­ta at­trib­uted to dif­fer­ences in base­line mea­sures be­tween the old­er chil­dren of the place­bo and treat­ment groups. That fail cut the biotech’s stock $SRPT by half, where it has hov­ered since. How­ev­er, in the sec­ond part of that Phase II study, in which 20 pa­tients who had re­ceived the place­bo treat­ment crossed over to get Sarep­ta’s gene ther­a­py, the pa­tients showed sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in the mo­tor abil­i­ties test that was al­so used in Part 1. Ear­li­er this month, Sarep­ta again post­ed pos­i­tive re­sults from oth­er parts of its gene ther­a­py pro­gram, show­ing that its gene ther­a­py sig­nif­i­cant­ly im­proved pa­tients’ mo­tor func­tion af­ter one year. But one pa­tient who had got­ten Sarep­ta’s gene ther­a­py ex­pe­ri­enced a case of my­ocardi­tis, or heart in­flam­ma­tion. No­tably, Pfiz­er’s Duchenne gene ther­a­py pro­gram al­so saw two cas­es of heart in­flam­ma­tion. Pre­vi­ous­ly, the two com­pa­nies, along with Sol­id Bio­sciences and Genethon, sug­gest­ed those cas­es may be tied to a “class ef­fect.” For its ac­cel­er­at­ed ap­proval, Sarep­ta plans to use both an in­ter­me­di­ate clin­i­cal end­point, the score on the func­tion­al mo­tor test af­ter one year, as well as a sur­ro­gate bio­mark­er, dy­s­trophin ex­pres­sion, a Sarep­ta spokesper­son told End­points News . Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect" Sarep­ta is no stranger to ac­cel­er­at­ed ap­provals, hav­ing got­ten them for its last three Duchenne mus­cu­lar dy­s­tro­phy treat­ments. Its first ac­cel­er­at­ed ap­proval came in 2016 for Ex­ondys 51, rife with con­tro­ver­sy. Sev­er­al FDA of­fi­cials at the time were against the ap­proval of the drug, with one call­ing it a “ sci­en­tif­i­cal­ly el­e­gant place­bo ,” but were over­shad­owed by Janet Wood­cock, who ad­vo­cat­ed for its ap­proval. “It set this in­ter­est­ing, slash con­cern­ing, prece­dent around dy­s­trophin as a val­i­dat­ed sur­ro­gate end­point, be­cause there’s not re­al­ly ev­i­dence of it ac­tu­al­ly hav­ing clear clin­i­cal as­so­ci­a­tion with things that are mean­ing­ful for as­sess­ing ben­e­fit (for) Duchenne mus­cu­lar dy­s­tro­phy,” Yale School of Med­i­cine pro­fes­sor Resh­ma Ra­machan­dran told End­points News . Ra­machan­dran not­ed that Ex­ondys 51’s ap­proval al­so set the prece­dent for Sarep­ta’s two fol­low­ing ac­cel­er­at­ed ap­provals. Ac­cord­ing to the gov­ern­ment’s clin­i­cal tri­als web­site, Sarep­ta’s con­fir­ma­to­ry tri­als for its three an­ti­sense oli­go treat­ments are set to fin­ish in 2024 — some eight years af­ter its first ap­proval. Sarep­ta’s lack of con­fir­ma­to­ry tri­al re­sults speaks to a big­ger pat­tern, in which com­pa­nies that get ac­cel­er­at­ed ap­provals drag their feet on con­fir­ma­to­ry tri­als or fail to do them at all, a prob­lem a bi­par­ti­san Sen­ate bill aims to re­form. In the com­ing weeks, Con­gress is like­ly to pass a bill that will broad­en the FDA’s au­thor­i­ty to en­sure con­fir­ma­to­ry stud­ies are un­der­way be­fore ac­cel­er­at­ed ap­proval is grant­ed. Sen­ate un­veils its ver­sion of ac­cel­er­at­ed ap­proval re­forms as bi­par­ti­san duo calls on FDA and PTO to work to­geth­er In­ter­est­ing­ly, in April, Sarep­ta moved up the time­line for Ex­ondys 51’s con­fir­ma­to­ry tri­al, dubbed MIS51ON, by about one and a half years, from ear­ly 2026 to late 2024. A Sarep­ta spokesper­son told End­points in an email the ear­li­er time­line re­flect­ed ad­di­tion­al tri­al sites. Pfiz­er is al­so de­vel­op­ing a gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy. While its Phase III tri­al was put on hold fol­low­ing a pa­tient death, the phar­ma gi­ant has since re­sumed the tri­al, it said in April. That tri­al is set to read out by late 2023. UP­DAT­ED: Pfiz­er can restart PhI­II tri­al of Duchenne gene ther­a­py, now on track for late-2023 FDA ap­proval re­quest No­tably, Sarep­ta is cur­rent­ly al­so run­ning a Phase III tri­al, dubbed EM­BARK, for its gene ther­a­py, and its end­point is the func­tion­al mo­tor test. How­ev­er, the es­ti­mat­ed com­ple­tion date for that Phase III tri­al cur­rent­ly stands at Oc­to­ber 2023, while Sarep­ta’s FDA ap­proval date could be ear­li­er in the year if it files for ac­cel­er­at­ed ap­proval this fall. The Sarep­ta spokesper­son con­firmed that Sarep­ta be­lieves EM­BARK would be the con­fir­ma­to­ry tri­al for SRP-9001’s ac­cel­er­at­ed ap­proval. “Buck­le up, the next year is go­ing to be an in­ter­est­ing de­bate on Sarep­ta and the time­line around FDA ac­tion, what de­fines ‘rea­son­ably like­ly to pre­dict’, and how EM­BARK read­out im­pacts the time­frame for ac­tion,” Sko­r­ney wrote.

Growing geriatric populace and surging prevalence of chronic conditions and disabilities are expected to spur market growth The global robotic rehabilitation & assistive technologies market size is expected to reach USD 4.08 billion by 2028 at a CAGR of 12.7%, according to a new report by Reports and Data. Substantial increase in market revenue growth can be attributed to the expanding geriatric population, rising prevalence of congenital physical disabilities, growing burden of acute and chronic diseases, and increasing incidences of stroke, paralysis, or spinal injuries. Other major factors accountable for market revenue growth are rising applications of computational intelligence in assistive technology, surging demand for advanced robotic prostheses, and rapid incorporation of rehabilitation robotics and assistive technologies across hospitals, clinics, and palliative care facilities. Rising collaborations among research laboratories, academia, and leading industry players have led to path-breaking advances in assistive robotic technologies such as powered exoskeleton, which enables individuals with locomotor disabilities to move or walk better. For instance, a research team at Northern Arizona University led by Prof. Zach Lerner is currently working on the development of wearable exoskeleton devices to help disabled people improve their mobility. Robotic rehabilitation and assistive technologies are part of an advanced medical approach to restoring functional ability of the ill or disabled and improving their quality of life with the help of robotic devices. Rehabilitation robotics comprise robotic devices specifically designed to assist various sensorimotor functions that involve the use of hands, arms, or limbs. They help patients with daily repetitive tasks through the recovery process and also gather quantitative data about their performance and progress. These devices have proven to be most effective for individuals with motor function damage caused by stroke, severe functional disabilities like muscular dystrophy and Parkinson’s disease, cognitive or visual impairment, hearing loss, congenital disabilities, or wheeled mobility. Rehabilitation robotics is a specialized branch of biomedical engineering and extensively involves human-robot interaction for rehabilitation purposes. Several assistive technologies such as soft robotics and exoskeletons enhance the efficacy of rehabilitation therapies and ease patients’ lives, boosting their self-dependence. Access Free sample PDF Copy of the Report @ Some Key Highlights from the Report: Based on application, stroke is the leading segment and is expected to account for largest revenue share over the forecast period. Increasing prevalence of cardiovascular disorders, rising cases of locomotor disability caused by stroke worldwide, and growing usage of rehabilitation robotics in motor skill therapy and gait training are among the key factors driving revenue growth of this segment. Asia Pacific robotic rehabilitation & assistive technologies market is expected to register the fastest revenue growth rate over the forecast period. Market growth in the region is supported by escalating demand for advanced rehabilitation robotic technologies, booming geriatric population, and rising cases of physical disabilities. Presence of state-of-the-art healthcare infrastructure in the region and augmented healthcare expenditures further boost the Asia Pacific market growth. The most prominent companies in the global robotic rehabilitation & assistive technologies market are ReWalk Robotics, AlterG, Inc., Bionik Laboratories Corp., Ekso Bionics, Vincent Medical, DIH Technologies Corporation (Hocoma), CYBERDYNE, INC., Kinova, Inc., Rex Bionics Ltd., and Instead Technologies, Ltd. In September 2020, Bionik Laboratories Corp., a leading robotic rehabilitation & assistive technology solutions provider, sold its BIONIK InMotion® ARM/HAND Interactive Therapy System to the Center for Neurorestoration and Neurotechnology (CfNN). The latest purchase is a part of CfNN’s mission to offer innovative solutions to Veterans with neurological conditions to help them restore their physical function. Buy now your Exclusive copy of Report @ For the purpose of this report, the global robotic rehabilitation & assistive technologies market is segmented based on type, portability, application, and region: Type Outlook (Revenue, USD Billion; 2018-2028) Humanoid Rehabilitation Surveillance & Security Socially Assistive Others Portability Outlook (Revenue, USD Billion; 2018-2028) Standalone Mobile Application Outlook (Revenue, USD Billion; 2018-2028) Post-surgery Stroke Orthopedics Cognitive & Motor Skill Therapy Others Request for Custom Research @ Regional Outlook (Revenue, USD Billion; 2018-2028) North America U.S. Canada Mexico Europe Germany U.K. France BENELUX Rest of Europe Asia Pacific China Japan India South Korea Rest of the Asia Pacific Latin America Brazil Rest of Latin America Middle East & Africa Saudi Arabia U.A.E. 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