Have you ever watched a baby die of pertussis? I have. Several. This is what it looks like: A newborn baby can’t stop coughing. They can’t catch their breath and they can’t eat. They get dehydrated and stop peeing. They sometimes have pauses in their breathing. Parents are

Wish I were in person at #WMS2024 but so thankful @WorldMuscleSoc provides a high quality and affordable virtual option. Grateful to be able to learn without having to travel and leave my young children.

Thanks to our @ChildrensPhila Clinical In Vivo Gene Therapy group for sharing their unique expertise w/ scientists & clinicians across specialties at Sept. 20's symposium! The event highlighted evidence-based research + offered best practices for #AAV #GeneTherapy in children.

Last week to register for our symposium on the clinical practice of #AAV based #genetherapy. w/ @LindseyAGeorge @DeniseSabatino2 @doshib25 @suzmatMD

Register for a Sept. 20 in-person event at @ChildrensPhila, The Safe and Effective Practice of AAV #GeneTherapy! During this full-day seminar, you'll hear from ASGCT Board Member @LindseyAGeorge, Past President Beverly Davidson, @DeniseSabatino2, + more. https://t.co/Kkt0MR5M4i

Proud to collaborate on this piece led by @oabdulhamid and @pandaneuro. Decision to pursue Elevidys treatment requires individualized detailed counseling and consideration of potential risks / benefits given unknowns.

Very exciting news on $srpt #elevidys expansion for the #DMD community. As treating physicians, it’s important we maintain a high safety standard and ensure systems are in place for thorough monitoring and follow up.

Super proud to share our collaboration w/the Clinic for Special Children on early dual treatment in SMA. All 2 copy children, despite early tx, developed ultrasound changes. Dual tx was safe, additional data on efficacy needed.

What a great first day at #ASCGT2024, and had the chance to speak about the implementation of approved #genetherapy. Awesome to share the stage with my #cigt colleague @DrSamelsonJones who spoke about #AAV clinical trials. So proud of what we are building @ChildrensPhila.

Seeing this after a full day of #SMA clinic hurts my heart. My early treated pts are running and jumping. It’s unacceptable to deny tx. #zolgensma works & time is motor neurons. How dare an insurance company deny a lifesaving treatment for these sweet babies. @DGlaucomflecken

3) Thanks @ESilvermanMD for sending me this article!

2) He walked for 2+ hours on Halloween and didn't need the wagon once 3) He jumped for the first time 4) He's barely falling anymore. Even when the FDA does approve - then the battle w/ insurance starts. Believe patients, believe rare disease experts, and change the system.

(1/3) This 1000%. We must develop new systems to evaluate rare disease therapies - patients deserve access. #Elevidys works. Things families of my patients have told me since getting the drug: 1) He can walk up the stairs at night.

Another first-in-human #AAV #genetherapy success at @ChildrensPhila supported by our Clinical In Vivo Gene Therapy Group w/ @LindseyAGeorge @suzmatMD Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time

This is tragic - yet another instance of an insurance company blocking a rare disease family from access to a life-changing therapy. Give rare disease families a chance, and let doctors dictate treatment plans. #genetherapy.

Great news - this is promising, excited to see what protein expression looks like at the higher doses. Higher protein levels with less frequent dosing would be great for patients! #duchenne #dmd Dyne Therapeutics Announces Positive Initial Clinical Data

Great news - excited to see more data on this drug, and looking forward to the future where we have more tools to treat #dystrophin disease

For @WHYYThePulse, host @MaikenScott explored how our on-site production of CAR T-cell products at @ChildrensPhila could lower the high cost of #genetherapies. Read or listen to the story now on @whyy:

Important work - glad to see the full story published. #genetherapy Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial - The Lancet Neurology

Nothing like having this video come out while at a conference with @LHF_EndDuchenne who had a huge part in making this happen. Proud to care for this incredible family - thank you for sharing your story. #duchennestrong #genetherapy $Srpt