Jon

For Rick Steves, travel is the antidote to American isolationism The travel expert sat down with The Wall Street Journal to discuss how time abroad can help people understand different perspectives and bridge divides.

Writer Rick Steves has made a career of sharing travel tips. Now he recommends travelers steer clear of the U.S.

“To be honest, if somebody asked me, ‘Should I go to America?’ I’d say no,” he said in a discussion with The Wall Street Journal’s Ryan Knutson.

CBER Roundtable on Cell and Gene Therapy The Roundtable will provide you with an opportunity to share your thoughts on advancing the field of cell and gene therapy.

🔊 TOMORROW, June 5: Join @fda.gov’s Cell and Gene Therapy Roundtable featuring ASGCT Board Members Terry Flotte, Paula Cannon, and more, on the panel! Watch on YouTube from 9 a.m. to noon (ET).

Do not miss out on this opportunity to hear from top regulatory leaders! www.fda.gov/vaccines-blo...

“Musk came to Washington all Cybertrucks and chain saws, ready to destroy the bureaucracy,” write Ashley Parker and Michael Scherer. But as he winds down his service, “he has found himself isolated within the upper reaches of the Trump administration”: https://theatln.tc/ckySMUOS

Life-Changing New Gene Therapy For #Duchenne Muscular Dystrophy: "children who have received the medication have been able to stay active and the effects of the one time infusion do not seem to wear off" 😲💪 #Elevidys delandistrogene moxeparvovec @sarepta.com www.texaschildrens.org/content/well...

Sarepta Therapeutics Presents Data at the American Society of Gene & Cell Therapy Conference, Including Statistically Significant Functional Outcomes for 8- and 9-Year-Old Patients in New Data Analysis of EMBARK Part 2 05/16/25 9:00 AM EDT Significant functional benefits for 8- and 9-year-olds with Duchenne in Part 2 of the EMBARK study, contributing to the evidence of stabilization or slowing of disease progression in later childhood when muscle weakness typically progresses Statistically significant differences were observed on all key endpoints including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort

In the recent analysis of Part 2 of the EMBARK study, participants with Duchenne muscular dystrophy who had received a placebo in Part 1 and were aged 8 to 9 years (n=14) at crossover were included. At one year post ELEVIDYS treatment, there were between-group differences (least square means) on all key endpoints that were statistically significant, including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort. "The latest data from the EMBARK study highlighting motor function improvements in 8- and 9-year-old boys is encouraging and adds to the growing body of evidence supporting ELEVIDYS," said Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics at the University of Arkansas for Medical Sciences and Arkansas Children’s Hospital. "What stands out is that these patients were treated at an age when motor decline is typically expected in those with Duchenne. Yet, those who received ELEVIDYS demonstrated statistically significant and clinically meaningful functional improvements compared to external controls."

The results presented at ASGCT are from the ongoing analysis of results from Part 2 of EMBARK, which compared two-year outcomes from 63 participants against data from an external control group of untreated individuals with Duchenne. Results at two years post-treatment showed that individuals treated with ELEVIDYS had better outcomes in multiple motor function measures, compared to a well-matched external control group. Additionally, no new safety signals were observed in the EMBARK study over the two-year duration and, in a subset of patients (n=16), micro-dystrophin expression and sarcolemmal localization was sustained from Week 12 to Week 64. “This has been a significant year for our neuromuscular portfolio, with multiple, ongoing analyses and longer-term data on efficacy and safety presented for ELEVIDYS,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta Therapeutics. “Building on the topline EMBARK Part 2 data from earlier this year, we’re committed to sharing ongoing analyses as fast as possible. The one-year results of patients treated with ELEVIDYS at 8 to 9 years old provide evidence that those treated with gene therapy outperform those who don’t receive it at a critical point when more dramatic functional decline is expected.”

"Statistically significant differences were observed on all key endpoints; Significant functional benefits for 8 and 9-year-olds with Duchenne in Part 2 of the EMBARK study" #Elevidys delandistrogene moxeparvovec #Duchenne muscular dystrophy #GeneTherapy investorrelations.sarepta.com/news-release...

Sarepta Therapeutics Shares New Protein Expression and Safety Results from ENDEAVOR in Participants 2 Years Old at Time of Treatment - Treatment with ELEVIDYS for Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6) - Safety profile consistent with prior studies of ELEVIDYS and real-world experience CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 16, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Treatment with ELEVIDYS in the ENDEAVOR participants in cohort 6 who were 2 years old at the time of treatment (n=6), demonstrated mean expression of 93.87% of normal, as measured by western blot, and 79.9 percent dystrophin positive fibers (PDPF), as measured by immunofluorescence. The results were seen in biopsies taken 12 weeks after treatment. Safety in cohort 6 was consistent with clinical and real-world experience with ELEVIDYS. The most common adverse events were nausea and vomiting. Elevated liver enzymes were seen in two patients and resolved with steroid administration. “The strength of the biomarker results that we are seeing in younger patients is extremely encouraging and we have a meeting with U.S. FDA next month to discuss expanding the ELEVIDYS label to include younger patients,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta Therapeutics. “In addition to positive expression results, the safety profile in these patients is consistent with what we’ve seen in prior studies and in patients who have been prescribed treatment.” Sarepta had previously shared expression and safety from Cohort 4 of ENDEAVOR, in which participants were 3 years old at the time of treatment. In biopsies taken 12 weeks after treatment with ELEVIDYS, participants in Cohort 4 (n=7) demonstrated mean protein levels of 99.64 percent, as measured by western blot. More than 25 patients under the age of 4 have now been treated in our clinical studies.

Protein sure is a hot topic these days! "Treatment with #ELEVIDYS (delandistrogene moxeparvovec) for #Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6)" 💪💪💪 #GeneTherapy investorrelations.sarepta.com/news-release...

Approval Information Product Name: ELEVIDYS® Intravenous Infusion Generic Name: delandistrogene moxeparvovec Contraindication and Prohibition (partial excerpt): Patients do not have a deletion of any portion or the entirety of exon 8 and/or exon 9 in the dystrophin gene Efficacy or Effects: Duchenne muscular dystrophy Indicated for patients who meet all of the following criteria: Patients who are negative for anti-AAVrh74 antibodies Ambulatory patients Patients aged 3 years or older and younger than 8 years

Approval Conditions and Time Limit: [Approval Conditions] During the period until the reapplication for marketing approval of this product after conditional and time-limited approval, post-marketing approval condition evaluation shall be conducted through clinical trials aimed at confirming the long-term efficacy and safety of this product, as well as post-marketing surveillance targeting all cases in which this product is used. Necessary measures shall be taken, including dissemination of proper use guidelines developed in cooperation with relevant academic societies, to ensure that physicians with sufficient knowledge and experience in Duchenne muscular dystrophy use this product in accordance with the "Efficacy or Effects" and "Dosage and Administration" after thoroughly acquiring knowledge of the clinical trial results and adverse events of this product, at medical institutions with established systems for treating Duchenne muscular dystrophy. Necessary measures shall be taken, including dissemination of the usage regulations, to ensure that this product is used in compliance with the Type 1 Use regulations approved under the "Act on the Conservation and Sustainable Use of Biological Diversity through Regulations on the Use of Living Modified Organisms (Act No. 97 of 2003)." [Time Limit] 3 years [Reference] Chugai Files Elevidys (SRP-9001) as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan (Press release by Chugai issued on August 14, 2024) https://www.chugai-pharm.co.jp/english/news/detail/20240814150000_1091.html Roche Announces Outcomes for EMBARK Study of Elevidys in Duchenne Muscular Dystrophy (Press release by Chugai issued on October 31, 2023)

Chugai Receives Regulatory Approval for #Elevidys (delandistrogene moxeparvovec) as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan 💪💚https://www.chugai-pharm.co.jp/english/news/detail/20250513181500_1160.html

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How Gene Therapy Is Changing Logan’s Life - Ivanhoe Broadcast News, Inc. Duchenne Muscular Dystrophy, or DMD, is a rare and fatal genetic disease that primarily affects boys, slowly robbing them of their ability to walk, breathe, and live independently. But now there’s a b...

How Gene Therapy Is Changing Logan’s Life #Duchenne muscular dystrophy #RareDisease #Elevidys delandistrogene moxeparvovec #GeneTherapy
www.ivanhoe.com/family-healt...

Watch: Sarepta Therapeutics - Duchenne Gene Therapy Update (Webinar Recording) Sarepta Therapeutics recently joined PPMD for a community webinar to share the latest updates on their gene therapy program. In addition to program updates, Dr. Chet Villa, cardiologist at Cincinnati ...

"Parent Project Muscular Dystrophy (PPMD)
Webinar recording:
@sarepta.com recently joined PPMD to share the latest updates on their #genetherapy program. Watch the recording:" www.parentprojectmd.org/watch-sarept... #Duchenne muscular dystrophy #Elevidys delandistrogene moxeparvovec

Welcome! You are invited to join a webinar: Sarepta Therapeutics: Duchenne Gene Therapy Update. After registering, you will receive a confirmation email about joining the webinar. Join us on Wednesday, April 23, at 1:00 PM ET for a community webinar with Sarepta Therapeutics as they share the latest updates on their gene therapy program. In addition to program updates, Dr. Chet...

Parent Project Muscular Dystrophy (PPMD): Sarepta Therapeutics: Duchenne Gene Therapy Update: Apr 23, 2025 12:00 PM CDT (Zoom) #Elevidys delandistrogene moxeparvovec parentprojectmd-org.zoom.us/webinar/regi...

HANS 006 2025 learning lessons from the saddest outcomes YouTube video by Rare Diseases with Dr. Hans

"Just over 3 weeks ago, Sarepta reported that a young man passed away after receiving their gene therapy for Duchenne Muscular Dystrophy. As a rare disease Dad, physician, and teammate in drug development, I want to share my 2 cents in the hopes it helps." #Elevidys www.youtube.com/watch?v=BjDm...

😢

Dr. Arif Khan | Pediatric Neurologist on Instagram: "Gene therapy for children with Duchenne muscular dystrophy. As a young Pediatric Neurologist 15 years ago, I never imagined that one day this would... 49 likes, 4 comments - dr.arif.khan on April 13, 2025: "Gene therapy for children with Duchenne muscular dystrophy. As a young Pediatric Neurologist 15 years ago, I never imagined that one day this wo...

Dr. Arif Khan - Fakeeh University Hospital: "Duchenne Musküler Distrofisi olan çocukları için gen terapisi arayan Türkiye'deki ebeveynlere mesajım." #Elevidys (delandistrogene moxeparvovec) #Duchenne muscular dystrophy #GeneTherapy #RareDisease www.instagram.com/dr.arif.khan...

Trump bouncing back and forth on tariffs and RFK on MMR vaccines are THE telltale signs of when the grift is unable to reconcile with reality.

The grift and grifter might change, but the behavior always stays the same.

Ex-DOJ Official to Reporters: 'There Are 2 Things Left – The Courts and You' - National Press Foundation Chuck Rosenberg has served at virtually every level, across Republican and Democratic administrations. This is like nothing he's ever seen.

Thank you to journalists who choose "to get paid less than you would anywhere else, to work harder than you would anywhere else, and to get yelled at more than you would anywhere else in a time when our institutions are faltering." -Chuck Rosenberg, ex DOJ nationalpress.org/topic/non-pa...

Former top FDA official warns RFK Jr. against giving "false hope" on autism "You can be incredibly supportive of people, but giving them false hope is wrong," said Dr. Peter Marks.

Former top FDA official warns RFK Jr. against giving "false hope" on autism

Former top health official criticizes RFK Jr. for downplaying measles deaths Dr. Peter Marks said that the deaths of unvaccinated children is "just not acceptable."

The former top vaccines official at the Food and Drug Administration criticized Health and Human Services Secretary Robert F. Kennedy Jr. for downplaying the deaths of unvaccinated children from measles, amid this year's record outbreak of the virus.

Nearly half the FDA’s $7 billion budget comes from fees collected from drug, device and tobacco companies. The agency uses the money to hire thousands of staffers to quickly and efficiently review new products. For example, about 70% of the FDA’s drug program is financed by user-fee agreements, which must be reauthorized by Congress every five years. But the agreements stipulate that if FDA’s federal funding falls below set levels, companies are no longer required to pay and, in some cases, can claw back their money. The threshold requirements are designed to ensure Congress continues funding FDA, rather than relying entirely on the private sector.

apnews.com/article/fda-... @apnews.com

Study: Who falls for misinformation? www.msn.com/en-us/news/t... via @profsanderlinden.bsky.social et al.

- Generation Z,
- non-male
- less educated
- more conservative

"...extreme conservatives' perceived ability showed little relation to their actual misinformation discernment."

nooooo c'mon guys, never give up, never surrender! #USAUSAUSA 😢 thehill.com/homenews/sen...

😂😅😂

Health secretary RFK Jr. endorses the MMR vaccine — stoking fury among his supporters Prominent anti-vaccine activists lined up on social media to denounce the move.

Prominent anti-vaccine activists lined up on social media to denounce the move. By @gbrumfiel.bsky.social

Americans Will Pay the Price for Reckless Tariffs | National Review As a share of the economy, President Trump’s executive order is likely the largest peacetime tax increase in U.S. history.

As a share of the economy, President Trump’s executive order on tariffs is likely the largest peacetime tax increase in US history.

trib.al/oWa97e1

Sarepta Therapeutics Provides Update on ELEVIDYS 04/04/25 8:44 AM EDT CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 4, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy. Following the safety update on acute liver failure that was issued on March 18, European Union (EU) reference member country authorities requested that the independent data monitoring committee (DMC) meet to review the adverse event. While the analysis is being finalized, recruitment and dosing in certain clinical studies of ELEVIDYS are temporarily halted. The independent DMC met on April 3 and concurred that based on the totality of evidence, the overall benefit-risk profile remains favorable to continue dosing in the paused clinical trials without changes to the study protocols. At the request of EU regulators, Sarepta and Roche will submit this information in a response to the temporary halt within a week. Evaluation of the submission and the subsequent decision for lifting the temporary halt will follow the EU regulatory process. The clinical studies affected by the temporary halt are Study SRP-9001-302 (ENVOL), Study SRP-9001-303 (ENVISION) and Study SRP-9001-104. Monitoring and data collection for already-enrolled participants continues, and we do not anticipate a material impact on the timeline for these studies.

Sarepta Therapeutics @sarepta.com Provides (safety) Update on #ELEVIDYS (delandistrogene moxeparvovec) #Duchenne muscular dystrophy #GeneTherapy #RareDisease investorrelations.sarepta.com/news-release...

all that money was woke, you see

Rare Disease Researchers Warn NIH Budget Cuts Could Threaten Progress and Endanger Lives With a $48 billion annual budget, the US National Institutes of Health is the world’s largest public funder of biomedical research.

An atmosphere of grave concern is gripping the #RareDiseaseCommunity as massive budget cuts & layoffs at the NIH endanger tens of millions of dollars in grants to study everything from Duchenne muscular dystrophy to idiopathic pulmonary fibrosis @mda.org 🧪♿
www.rarediseaseadvisor.com/features/nih...