Working together to bring individualized genetic medicines safely and rapidly to patients worldwide
- Tweets 31
- Following 2
- Followers 132
- Likes 0
Individualized medicine provides new opportunities for targeted therapies for rare disease patients, but not all individuals affected by rare disease are eligible for tailored N=1 approaches.
1
1
1
393
Join us at the next N=1 Collaborative seminar led by Marlen Lauffer to discuss recommendations for evaluation of disease causing variants.
Register: bit.ly/3Lq6J3I
1
164
Join us for a case study on Monday, September 11, at 12:30 pm US EDT to hear about ASO treatment for SCN2A-encephalopathy, allele-specific vs non-specific, with Heather Olson and Elizabeth Berry-Kravis.
Register here: bit.ly/487WDhC
2
2
4
562
For N-of-1/few treatments in rare genetic disease, while antisense oligos may be the initial therapeutic platform of choice, CRISPR holds huge promise for the future.
1
1
12
2,187
@UrnovFyodor and Manar Zaghlula from the Innovative Genomics Institute will join the @N1Collaborative Seminar Series on Monday 8/28 at 12:30-1:30p EST to share a framework and recommendations for enabling affordable, accessible bespoke CRISPR treatments.
1
2
227
Join us for a case study on Monday, July 31, at 12:30 pm US EDT to hear about a novel antisense oligonucleotide for CMT2S, a rare subtype of Charcot-Marie-Tooth Disease, with Sandra Smieszek of Vanda Pharmaceuticals.
1
3
197
Our N=1 Collaborative seminars are open to everyone and we encourage questions and discussion!
Join Live: bit.ly/3rBemxh
64
Join Andrew Lo, @MIT Sloan Professor of Finance, this Monday for a dynamic seminar hosted by the @N1Collaborative. We will discuss the portfolio model in rare disease therapeutics, the role of philanthropy, and reimbursement considerations in the individualized medicine context.
1
2
167
What if coding personalized ASO therapies only required the press of a button? Join us Monday, July 10, at 12:30pm US EDT, and Vanessa Almendro of @DanaherCorp to discuss the paradigm shift needed to make therapeutic interventions for rare genetic diseases accessible to patients.
1
1
3
390
Joining this discussion will be a panel of innovators from Integrated DNA Technologies, Cytiva, and La Jolla Labs Inc.
Join Live: bit.ly/46wLhTT
86
Join Emil Kakkis, CEO of Ultragenyx & Richard Finkel, MD, Director, Center for Experimental Neurotherapeutics, St. Jude Children's Hospital, at the @N1Collaborative seminar Monday, June 5 at 12:30 PM EDT to discuss how to think about outcome measures for individualized medicines.
1
6
1,094
The talk will include novel approaches like the Multi-Domain Responder Index, as well as how to incorporate biomarkers.
Join Live: bit.ly/3ITqcJ3
@stopbatten @winstonxyan @timyu
84
Identifying patients who could benefit from individualized ASOs requires consideration of the underlying variant, the disease and the patient.
1
1
116
Join us on Monday, May 22, at 12:30pm US EDT, for the next @N1Collaborative seminar to discuss this critical topic with Dr. Matthis Synofzik and Dr. Rebecca Schüle from University of Tuebingen
Join live: bit.ly/3BG7j8u
1
68
Join us today, May 3, at 2pm US EDT, for an @N1Collaborative seminar. Ali Fatemi, MD, MBA of Kennedy Krieger Inst. & Beth McGinn of CureLBSL highlight work toward an N-of-small ASO treatment for #LBSL, an ultrarare neurodegenerative disorder.
JOIN LIVE: bit.ly/3oZRxSB
1
93
The @N1Collaborative invites interested investigators to join our Seminar Series on Individualized Medicines this Monday, April 24th, 12:30-1:30pm US ET to discuss Regulatory Submissions and Institutional Implementation.
1
1
90
Margot Cousin from @MayoClinic, Roger Paxton from @ChildrensColo and Catherine Douthwright from @UMassChan will present several illustrated examples including protocols, informed consent, and pre-IND & IND applications.
1
65
We welcome and encourage your thoughts in the discussion that will follow. Mark your calendars! JOIN LIVE this Monday - bit.ly/41sgTa0
49