Latest news with #DM1
Yahoo
30-05-2025
- Business
- Yahoo
PepGen price target lowered to $1 from $3 at BofA
BofA lowered the firm's price target on PepGen (PEPG) to $1 from $3 and keeps an Underperform rating on the shares after the company announced it will be discontinuing development of Duchenne muscular dystrophy programs following 'disappointing' 10mg/kg data for PGN-EDO51. PepGen will now focus developmental efforts on its DM1 program, which the firm thinks 'still needs significant de-risking,' the analyst tells investors. The firm updated its model for Q1 results, cash and share count and removed value for DMD from its model based on the discontinuation. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See Insiders' Hot Stocks on TipRanks >> Read More on PEPG: Disclaimer & DisclosureReport an Issue PepGen to discontinue DMD programs after PGN-EDO51 did not achieve target PepGen price target lowered to $8 from $14 at H.C. Wainwright PepGen Inc.'s Strategic Shift Amidst DMD Program Discontinuation and Competitive Challenges PepGen Inc. Discontinues DMD Programs After Trial Results PepGen appoints Kasra Kasraian as CTO Sign in to access your portfolio
Yahoo
30-05-2025
- Business
- Yahoo
PepGen to discontinue DMD programs after PGN-EDO51 did not achieve target
PepGen (PEPG) announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy patients amenable to exon 51 skipping, the company will focus on advancing its 'promising' myotonic dystrophy type 1 program currently in Phase 2 clinical development. The company is voluntarily discontinuing development of PGN-EDO51 and intends to wind down all DMD-related research and development activities. In the 10 mg/kg cohort of the CONNECT1 study, PGN-EDO51 increased exon 51 skipped transcripts to 4.26%; however, total dystrophin only increased to 0.59% of normal levels. The safety profile of PGN-EDO51 continued to be generally favorable and all treatment-related adverse events were mild in nature. No serious adverse events were reported in the study. 'We are disappointed by the dystrophin results observed in the 10 mg/kg dose cohort in CONNECT1, as it was our hope that we could improve upon existing therapies for patients in a more profound way,' said James McArthur, PhD, President and CEO of PepGen. 'As we wind down our DMD program, we would like to thank the patients, families, caregivers, investigators and study staff for their support and participation in this research. I also want to acknowledge our team's hard work and commitment to advancing new potential treatments for DMD patients.' PepGen continues to expect to report data from its FREEDOM-DM1 15 mg/kg cohort in patients with DM1 during the second half of 2025. FREEDOM is a Phase 1 single ascending dose, randomized, placebo-controlled clinical trial, with endpoints including safety, 28-day splicing correction and functional benefit measures. The company also continues to expect to report data from the 5 mg/kg cohort of its FREEDOM2-DM1 study in DM1 patients in the first quarter of 2026. FREEDOM2 is a Phase 2 multiple ascending dose, randomized, placebo-controlled clinical trial, with endpoints following four doses that include safety, splicing correction and functional benefit measures. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on PEPG: Disclaimer & DisclosureReport an Issue PepGen price target lowered to $8 from $14 at H.C. Wainwright PepGen Inc.'s Strategic Shift Amidst DMD Program Discontinuation and Competitive Challenges PepGen Inc. Discontinues DMD Programs After Trial Results PepGen appoints Kasra Kasraian as CTO PepGen Inc.'s Promising Advances in DMD and DM1 Programs Justify Buy Rating Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
28-05-2025
- Business
- Business Wire
PepGen to Focus on Development of Promising DM1 Program Following 10 mg/kg PGN-EDO51 Update
BOSTON--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies, today announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping, the Company will focus on advancing its promising myotonic dystrophy type 1 (DM1) program currently in Phase 2 clinical development. The Company is voluntarily discontinuing development of PGN-EDO51 and intends to wind down all DMD-related research and development activities. In the 10 mg/kg cohort (n=4) of the CONNECT1 study, PGN-EDO51 increased exon 51 skipped transcripts to 4.26% (a mean increase of 3.5%); however, total dystrophin only increased to 0.59% of normal levels (a mean increase of 0.36%). The safety profile of PGN-EDO51 continued to be generally favorable and all treatment-related adverse events were mild in nature. No serious adverse events were reported in the study. 'We are disappointed by the dystrophin results observed in the 10 mg/kg dose cohort in CONNECT1, as it was our hope that we could improve upon existing therapies for patients in a more profound way,' said James McArthur, PhD, President and CEO of PepGen. 'As we wind down our DMD program, we would like to thank the patients, families, caregivers, investigators and study staff for their support and participation in this research. I also want to acknowledge our team's hard work and commitment to advancing new potential treatments for DMD patients.' Paul Streck, MD, MBA, Executive Vice President, Head of R&D of PepGen continued, 'PGN-EDODM1, PepGen's investigational drug in development for DM1, has already demonstrated robust target engagement after a single 10 mg/kg dose in patients that resulted in mean mis-splicing correction of 29% with a favorable emerging safety profile as of February 24, 2025, the most recent safety update. Mis-splicing is the primary driver of DM1 pathology and currently patients have no approved treatment options for this disabling, life-shortening disorder. Going forward, we will focus our resources on advancing the Company's ongoing DM1 clinical program along with our research pipeline.' PepGen continues to expect to report data from its FREEDOM-DM1 15 mg/kg cohort in patients with DM1 during the second half of 2025. FREEDOM is a Phase 1 single ascending dose, randomized, placebo-controlled clinical trial, with endpoints including safety, 28-day splicing correction and functional benefit measures. The Company also continues to expect to report data from the 5 mg/kg cohort of its FREEDOM2-DM1 study in DM1 patients in the first quarter of 2026. FREEDOM2 is a Phase 2 multiple ascending dose, randomized, placebo-controlled clinical trial, with endpoints following four doses that include safety, splicing correction and functional benefit measures. About PGN-EDODM1 PGN-EDODM1, PepGen's investigational candidate in development for the treatment of myotonic dystrophy Type 1 (DM1), utilizes the Company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 is designed to directly address the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the dystrophia myotonic protein kinase (DMPK) transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, disrupting the binding between the CUG repeat expansion and MBNL1. DM1 is a progressively disabling, life-shortening genetic disorder. DM1 is estimated to affect 40,000 people in the United States, and over 74,000 people in Europe. The U.S. Food and Drug Administration has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1. About PepGen PepGen is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen's EDO platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, we are generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases. For more information, please visit Follow PepGen on LinkedIn and X. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as 'aims,' 'anticipates,' 'believes,' 'could,' 'estimates,' 'expects,' 'forecasts,' 'goal,' 'intends,' 'may,' 'plans,' 'possible,' 'potential,' 'seeks,' 'will,' and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the therapeutic potential and safety profile of our product candidates, including PGN-EDO51 and PGN-EDODM1, our plans to discontinue our DMD-related research and development activities, including our CONNECT1-EDO51 and CONNECT2-EDO51 Phase 2 clinical trials, based on results from the CONNECT1 trial, the design and conduct of clinical trials, including our FREEDOM-DM1 Phase 1 trial and FREEDOM2-DM1 Phase 2 trial, and the expected timing for additional data from our FREEDOM Phase 1 trial and initial data from our FREEDOM2 Phase 2 trial. Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release 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 related to: delays or failure to successfully initiate or complete our ongoing and planned development activities for our product candidates, including PGN-EDODM1; our ability to enroll patients in our clinical trials, including FREEDOM and FREEDOM2; that our interpretation of clinical and preclinical study results may be incorrect, or that we may not observe the levels of therapeutic activity in clinical testing that we anticipate based on prior clinical or preclinical results, including for PGN-EDODM1; our product candidates, including PGN-EDODM1, may not be safe and effective or otherwise demonstrate safety and efficacy in our clinical trials; adverse outcomes from our regulatory interactions, including delays in regulatory review, clearance to proceed or approval by regulatory authorities with respect to our programs, including clearance to commence planned clinical studies of our product candidates, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our FREEDOM and FREEDOM2 clinical trials; changes in regulatory framework that are out of our control; unexpected increases in the expenses associated with our development activities or other events that adversely impact our financial resources and cash runway; and our dependence on third parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning PepGen's programs and operations are described in our most recent annual report on Form 10-K and quarterly report on Form 10-Q that are filed with the SEC. PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. This release discusses PGN-EDO51 and PGN-EDODM1, investigational therapies that have not been approved for use in any country and is not intended to convey conclusions about their efficacy or safety. There is no guarantee that PGN-EDODM1 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval.
Yahoo
21-04-2025
- Business
- Yahoo
Avidity Biosciences Receives Orphan Drug Designation in Japan for Delpacibart Etedesiran (del-desiran) for Treatment of Myotonic Dystrophy Type 1
Del-desiran first-ever investigational treatment for DM1 to receive Orphan Drug designation in Japan SAN DIEGO, April 8, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation (ODD) to delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1), an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. Del-desiran is the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. Del-desiran has also received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). "This decision by MHLW further reinforces the significant potential of del-desiran to address the root cause of DM1 and the urgent need to bring an approved therapy to the many people impacted by this devastating rare disease in Japan and around the world," said Steve Hughes, M.D., chief medical officer at Avidity. "We are very encouraged by del-desiran data reported from the MARINA and MARINA-OLE studies thus far, demonstrating favorable long-term safety and tolerability, reversal of disease progression, and consistent and durable improvements in multiple clinical endpoints. Looking ahead, there are multiple key milestones this year as we complete enrollment in the phase 3 HARBOR trial and continue to advance our global commercialization preparations to potentially deliver the first globally approved drug for people living with DM1." Avidity has aligned with global regulators on the registrational path for del-desiran for the treatment of DM1, which informed the design of the ongoing Phase 3 HARBOR™ study. Avidity expects to complete participant enrollment in the Phase 3 HARBOR study in mid-2025 and submit marketing applications starting 2026 in the U.S., European Union and Japan. Japan's MHLW grants Orphan Drug designation to drugs in development for the treatment of diseases that affect fewer than 50,000 patients in Japan and for which there is a high unmet medical need. An investigational therapy is eligible to qualify for Orphan Drug designation if there is no approved alternative treatment option or if there is high efficacy or safety expected compared to existing treatment options. Orphan Drug designation provides certain benefits, including prioritized consultation regarding clinical development, reduced consultation fees, tax incentives, priority review of applications, reduced application fees, and extended registration validity period. About the Phase 3 HARBOR™ TrialThe global Phase 3 HARBOR™ trial is a randomized, placebo-controlled, double blind pivotal study designed to evaluate del-desiran in approximately 150 people (age 16 and older) living with DM1. The trial will be conducted at approximately 40 sites globally. Patients will be administered either del-desiran or placebo (1:1) every eight weeks. HARBOR is designed to assess multiple key functional aspects of DM1. The primary endpoint is video hand opening time (vHOT), a measurement of myotonia, which is a hallmark symptom of DM1. Key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. All study participants, regardless of whether they receive active treatment or placebo, will have the option to enroll into an open-label extension trial. For more information about the HARBOR trial, visit the HARBOR study website or visit and search for NCT06411288. About the Phase 2 MARINA-OLE™ StudyMARINA-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of del-desiran in participants with DM1 who were previously enrolled in the MARINA® Phase 1/2 trial. This trial will continue to evaluate the safety, tolerability, PK, PD, and efficacy of del-desiran in participants enrolled in the randomized, placebo-controlled, Phase 1/2 MARINA clinical trial. Participants enrolled in the MARINA-OLE study receive quarterly doses of del-desiran regardless of whether they received active treatment or placebo in the MARINA study. The total duration of active treatment with del-desiran in the MARINA-OLE study is approximately 24 months. Once patients have completed active treatment, there will be a nine-month safety follow-up period. Avidity may extend active treatment beyond 24 months at a future timepoint. For more information on this study click here or visit and search for NCT05479981. About Del-desiranDel-desiran, Avidity's lead product candidate utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. Del-desiran consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. Del-desiran is currently being assessed in the global Phase 3 HARBOR™ trial and in the ongoing MARINA-OLE™ trial in people with DM1. Long-term data from the MARINA-OLE trial showed reversal of disease progression in people living with DM1 across multiple endpoints including video hand opening time (vHOT) as a measure of hand function and myotonia, muscle strength and activities of daily living when compared to END-DM1 natural history data. Del-desiran has received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). About Myotonic Dystrophy Type 1Myotonic dystrophy type 1 (DM1) is an underrecognized, autosomal dominantly inherited, progressive and often fatal disease caused by a triplet-repeat in the DMPK gene, resulting in a toxic gain of function mRNA. The disease is highly variable with respect to severity, presentation and age of onset, however all forms of DM1 are associated with high levels of disease burden and may cause premature mortality. DM1 primarily affects skeletal and cardiac muscle, however patients can suffer from a constellation of manifestations including myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment. Currently, there are no approved treatments for people living with DM1. About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare neuromuscular diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit and engage with us on LinkedIn and X. Forward-Looking StatementsAvidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: planned marketing applications for del-desiran in the U.S., European Union and Japan, and the timing thereof; Avidity's plans to become a global commercial organization and the status of its commercialization efforts; the characterization of data associated with del-desiran in the MARINA and MARINA-OLE studies, the conclusions drawn therefrom, and the impact of such data on the advancement of del-desiran and its ability to treat DM1; the designs, goals, statuses and enrollment levels of, and plans for, the MARINA-OLE and HARBOR studies; Avidity's platform, planned operations and programs; and Avidity's cash position and runway. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: Avidity may not realize any benefit from Orphan Drug designation of del-desiran by global health authorities; the data and results produced in Avidity's ongoing studies of del-desiran as of the most recent respective cutoff dates may not be indicative of final results, may not support a BLA submission, may not be satisfactory to the MHLW and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; even if approved, Avidity may not be able to execute any successful product launches; Avidity's efforts to build a global commercial organization may be unsuccessful; unexpected adverse side effects to, or inadequate efficacy of, del-desiran that may delay or limit its development, regulatory approval and/or commercialization; later developments with the MHLW and other global regulators that could be inconsistent with the feedback received to date; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven and may not produce any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of the MARINA-OLE and HARBOR studies; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; legislative, judicial and regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2024. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Contact:Kat Lange(619) 837-5014investors@ Media Contact:(619) 837-5016media@ View original content to download multimedia: SOURCE Avidity Biosciences, Inc. Sign in to access your portfolio
Daily Mail
21-04-2025
- Health
- Daily Mail
Doctors discover new cause of autism: 'Fourteen times more likely'
Autism may be caused by a little-known genetic condition, experts say. They've found children with myotonic dystrophy type 1 (DM1) are also 14 times more likely to develop autistic spectrum disorder. DM1 is a disorder people inherit from their parents that causes progressive muscle weakness, fatigue, and a host of cognitive issues. Experts believe it may also affect how the brain develops and processes information in early life, potentially altering neural pathways linked to communication, behavior, and social interaction — hallmarks of autism. The discovery could help unlock part of the mystery around how autism develops, providing vital clues to its biological roots. DM1 is caused by a faulty gene, and now researchers believe that gene may also play a role in autism. The researchers said the findings are a step closer to understanding autism not just as a spectrum — but as something with specific, traceable origins. And crucially, it raises hopes for more targeted support for patients with both conditions and treatment that focuses on repairing damaged genes. However, the team also emphasized DM1 is significantly more rare than autism, and not everyone with DM1 will be diagnosed with autism. In DM1, DNA strands in a gene called DMPK repeat, a process called tandem repeat expansions (TREs), causing the gene to not function properly. For people with DM1, this leads to symptoms like progressive muscle weakness and involuntary movements. The functional errors lead to protein imbalances affecting other genes responsible for brain function. The researchers said these gene impairments may cause people with DM1 to develop signs of autism like repetitive movements, a lack of coordination and sensory issues. While autism affects about 7million Americans, only about 140,000 are diagnosed with DM1. The study authors, from the University of Nevada Las Vegas (UNLV), said the findings could make it easier to diagnose autism in people with conditions like DM1 and lead to treatments to repair the damaged genes. Dr Ryan Yuen, study author and senior scientist in the Genetics & Genome Biology program at the Hospital for Sick Children in Las Vegas, said: 'Our findings represent a new way to characterize the genetic development of autism. 'By identifying the molecular pathway behind this connection, we can begin to investigate new approaches to ASD diagnosis and the development of precision therapies that release these proteins back into the genome.' This means essential proteins sapped away from DNA could be added back, repairing faulty genes and preventing further errors from appearing. Meanwhile, another new study from researchers in China found a non-invasive brain stimulation treatment may improve certain autism signs like trouble sleeping and issues with social interaction. The treatment, called transcranial pulsed current stimulation (tPCS) involves sending electrical impulses through electrodes that are placed on a patient's scalp. The signals coming through the electrodes are thought to increase brain activity in certain areas. The team found children ages three to 14 who received 20 tPCS sessions over four weeks had 'significant' improvements in sleep, language, sensory issues and socialization. Both studies come as the latest CDC data shows autism is on the rise in the US, affecting one in 31 children. This is a staggering increase from one in 150 in the early 2000s. While many experts believe the rise is due to better screening and diagnostics, health authorities like Robert F Kennedy Jr believe environmental factors like pesticides, food additives and ultrasound scans could be to blame. In the UNLV study, published in Nature Neuroscience, researchers analyzed RNA in 38 gene sets from people with and without autism. RNA is a key molecule that helps cells build proteins and function properly. The team found that when the DMPK gene responsible for DM1 repeats, it creates 'toxic RNA' that binds to proteins involved in DNA production during brain development. That 'toxic RNA' depletes the protein and prevents it from binding the other RNA molecules, causing a protein imbalance and errors in other surrounding genes. Dr Yuen said: 'TREs are like a sponge that absorbs all these important proteins from the genome. Without this protein, other areas of the genome don't function properly.' The researchers noted both DM1, the muscle-weakening condition, and autism can both be caused by repetitions in the DMPK gene. Dr Lukasz Sznajder, a research lead and assistant professor at the University of Nevada Las Vegas (UNLV), said: 'A variation really stood out to me that we see in rare neuromuscular disease. 'This is how we started connecting the dots. We found a molecular link, or overlap, which we believe is the core of causing autistic symptoms in children with myotonic dystrophy.' DM1 leads to muscle weakness around the limbs and, as it progresses, vital organs like the heart and lungs. This leads to abnormal heart rhythms and breathing issues. Symptoms typically appear in adolescence or young adulthood and start with weakness around the muscles in the face, neck, fingers and ankles. The condition affects 140,000 Americans and the life expectancy is around 48 to 55 years. The study authors said more research is needed connecting DM1 to autism, but the group is planning to look at if these DNA errors are occurring in other genes that have been associated with autism.
