Frequently asked questions

SciFam is a four day, in-person, neuromuscular conference for stakeholders in congenital muscular dystrophy, nemaline myopathy, and titinopathy. While there will not be a virtual attendance option this year, we plan to host an online event sometime following the conference. Stay tuned for more information later this year. Dates • Friday, August 1: Afternoon Welcome Reception for all attendees, Evening Social Activities • Saturday, August 2: Affected/Family Plenary and Subtype-Specific Programming, Teen+Young Adult programming, Kids Camp, Evening Social Activities • Sunday, August 3: Affected/Family Plenary and Subtype-Specific Programming, Bonus Sessions (Quality of Life topics), Kids Camp, Poster Session for all attendees • Monday, August 4: Invitation Only Scientific Symposium, Evening Reception and Poster Session for Researchers & Clinicians • Tuesday, August 5: Invitation Only Scientific Symposium and Afternoon Poster Session for Researchers & Clinicians Agenda While specifics are still in development, the preliminary agenda can be found at: scifam.info/2025-agenda.(https://scifam.info/2025-agenda) Check back often for updates.

Scientific Symposium On Monday, August 4 - Tuesday, August 5, we will host the Scientific Symposium, this year scheduled to follow the Family Conference. This invitation-only meeting is a forum for researchers and clinicians to present cutting-edge findings, discuss emerging therapies, forge collaborations, and set research priorities for the five primary subtypes of congenital muscular dystrophy, nemaline myopathy, and titinopathies. This is a very high level meeting, that will lean heavily on scientific language and understanding (not lay-friendly). As an affected individual, family member, industry representative, or other stakeholder, you may request an invitation to join this meeting during registration. Seating is limited for those who are not researchers or clinicians working in the above mentioned conditions. Children under the age of 12 will not be permitted to join the meeting, and no childcare will be available on these days. As our presenters may share unpublished work, all Scientific Symposium attendees must sign a confidentiality statement before joining the meeting on both days, and participants may not take photographs of the presenters or their slides. Please respect this rule and those working to identify treatments for our community.

Conference Venue & Hotel Reservations Hilton Philadelphia at Penn's Landing 201 S Christopher Columbus Bl Philadelphia, PA 19106 USA All attendees wishing to book a standard non-accessible room (or rooms) must use the link below, created specifically for our event. Please do not book your accommodations with the conference venue separately as we are liable for filling a considerable number of rooms, and you won't receive our group discount. scifam.info/2025-hotel-reservations (https://scifam.info/2025-hotel-reservations) Availability is not guaranteed after July 5. Room rates are $199 per night, plus tax. Any charges made to your room for ancillary services are your responsibility, and will be charged to your method of payment upon check out. You may customize check-in and check-out dates and the number of beds (1K/2Q) during booking, and you may request adjoining rooms. If available, rooms may be booked up to 3 days before, and 3 days after the conference. Additional dates will need to be added separately by contacting the hotel directly. The only accessible rooms still available at the conference venue are King Bed with Accessible Tub. If you need an alternative room type, see below for other booking options. Below is a list of accessible room alternatives close to the conference venue. Accessible Room Alternatives Philadelphia Marriott Old City ((marriott.com/en-us/hotels/phlmo-philadelphia-marriott-old-city/overview)0.2 miles from the venue) One Dock St (215) 238-6000 12 (unspecified) accessible rooms, no shuttle. Penn’s View Hotel (pennsviewhotel.com)(0.3 miles from venue) 14 N Front St 1-215-922-7600 4 (unspecified) accessible rooms, no shuttle. Holiday Inn Express (ihg.com/holidayinnexpress/hotels/us/en/philadelphia/phlpl/hoteldetail)(0.6 miles from venue) 100 N Christopher Columbus Bl 1-215-627-7900 9 accessible rooms (3 with roll-in showers), no shuttle. Kimpton Hotel Monaco Philadelphia (monaco-philadelphia.com)(0.4 miles from venue) 433 Chestnut St 1-215-925-2111 4 accessible rooms (one with a roll-in shower), no shuttle. Renaissance Philadelphia Downtown Hotel (marriott.com/en-us/hotels/phlpr-renaissance-philadelphia-downtown-hotel)(0.4 miles from venue) 401 Chestnut St 1-215-925-0000 7 roll-in shower rooms, no shuttle. Wyndham Philadelphia Historic District (wyndhamhotels.com/wyndham/philadelphia-pennsylvania/wyndham-philadelphia-historic-district/overview)(0.5 miles from venue) 400 Arch St 1-215-923-8660 8 wheelchair accessible rooms (4 with roll-in showers), no shuttle.

Sponsored Attendees and Scholarships Cure CMD, A Foundation Building Strength, and Team Titin will provide a limited number of scholarships to affected individuals, caregivers, and families who plan to attend 2025 SciFam but need financial assistance to do so. If you are interested in applying for a scholarship, learn more and apply here.(https://scifam.info/2025-scholarship-app) Sponsored researchers and clinicians, as well as families who are awarded scholarships must still use this link (https://scifam.info/2025-hotel-reservations)to book hotel reservations. You will be asked to provide a method of payment at the time of booking, but your credit card will only be charged in the following instances: • If your arrival or departure dates are outside of your sponsored accommodation dates, you will be charged for those additional room nights. • Any ancillary charges made to your room are your responsibility to pay upon check out. • Reservations may be cancelled up to 3 days prior to your scheduled arrival. Cancellations after this date (or no-shows) will incur a charge for the first night's stay on your method of payment. This charge will be made directly by the venue. If you have any questions, don't hesitate to get in touch.(https://www.scifam.info/contact)

Registration Affected Community & Unsponsored Researchers/Clinicians • Adults (18+): $150 • Teens (13-17): $100 • Children (2-12): $50, waived if registering for Kids Camp • Infants (0-23 months): Free Industry or Commercial Entity Representatives • $450

Kids Camp • Programming and Activities for Children ages 2-12 for the duration of the Affected/Family Sessions (Saturday & Sunday ONLY). • Children under the age of 2 may participate in Kids Camp activities at no charge as long as they are accompanied by a personal attendant at all times. Kids Camp Attendants cannot be responsible for children under the age of 2. • Children must be registered for both days; you may not register for just one day of Kids Camp. There are no exceptions. • Personal attendants must be registered for the conference. • Kids Camp attendants will have some understanding about special needs related to a child with a neuromuscular condition. They will escort a child to the restroom, but will not assist with toileting related to mobility limitations. They will also not assist with feeding, suctioning, breathing treatments, or taking medication. • Children attending Kids Camp along with their own personal attendant will receive a discount. We'd also appreciate any volunteers willing to be a Kids Camp attendant, or to minimally assist Kids Camp attendants while performing personal attendant duties. Please contact us (https://www.scifam.info/contact)if you would like to offer your assistance. Kids Camp Pricing Unattended Children 1st Child: $300 2nd Child: $200 3rd/4th/5th Child (each): $150 Children Accompanied by a Personal Attendant 1st Child: $200 2nd Child: $100 3rd/4th/5th Child (each) $50

Friday Welcome Reception On Friday, August 1 from 4:00pm-6:00pm, we will host conference check-in and a free welcome reception for all attendees who plan to arrive that afternoon. Please let us know during registration whether you plan to attend so we can prepare for your arrival! If your arrival time is later on Friday or sometime throughout the weekend, you can visit the registration desk during conference hours to receive your badge and swag bag. Saturday Social Events On Saturday, August 2 from 7:30pm-9:30pm, we will host a variety of social opportunities. There is no charge to participate in these events, please let us know during registration whether you plan to participate. Meals All meals are optional, and there are a number of onsite and offsite alternatives. See pricing below should you wish to opt in. Meal pricing has been set by the venue. Saturday Dinner No Saturday evening meal will be provided, on or off site. We encourage you to explore the city and the many restaurants it has to offer. Be sure to check out our Philadelphia Visitor's Guide for some great options! Meal Pricing Breakfast (Saturday, Sunday, Monday, Tuesday) Adult (Age 13+): $49 Child (Ages 2-12): $29 Lunch (Saturday, Sunday, Monday, Tuesday) Adult (Age 13+): $64 Child (Ages 2-12): $39 Dinner (Friday, Sunday, Monday) Adult (Age 13+): $99 Child (Ages 2-12): $45

Submit a request (https://scifam.info/2025-meet-with-an-expert)to meet with a clinical or advocacy expert at SciFam to ask your generalized questions during a 15 minute personalized Q&A. This year, in addition to plenary and subtype-specific sessions on care (at which you are encouraged to ask generalized questions), we're offering SciFam attendees the opportunity to meet with an expert for a 15 minute, 1:1 session to ask a question or set of related questions specific to a single topic. Requirements • You must be of legal age of majority ("adult"), registering for a meeting on behalf of yourself as the affected individual, or your affected child. • You may request a meeting on behalf of an affected young adult without a parent or guardian present, but a young adult may not request a meeting directly. • You must be registered to attend the 2025 Scientific & Family Conference. • You or your affected loved one must have a preliminary or confirmed diagnosis of one of the following neuromuscular conditions: • Collagen 6 Related Dystrophy (Ullrich/Bethlem) • Dystroglycanopathy (WWS, MEB, Dystroglycan-related CMD or LGMD) • LAMA2-Related Dystrophy • LMNA-Congenital Muscular Dystrophy • Nemaline Myopathy • SELENON-Related Myopathy • Titinopathy Following submission of this form and before a meeting can be scheduled, you will receive an email asking you to provide proof of diagnosis via secure upload by submitting either a genetic report, or narrative summary from a physician. On this form, you must limit your inquiry to one topic and one type of expert. Your options are: • Neurologist • Pulmonologist/RT • Physiatrist/Physical Medicine & Rehabilitation • Nutritionist or Gastroenterologist • Orthopedic Surgeon • Physical therapist • Occupational Therapist • IEP/504 Education Plan Specialist • Case Management Support* Your meeting will be scheduled on Saturday or Sunday morning during the normal conference agenda, depending on the expert's availability. *Case Management Support: Courtney Jones from the Patient Advocate Foundation will offer 1:1 support for those seeking help with issues around insurance denials, healthcare access, and medical debt crises. Disclaimer Information provided during a personalized Q&A meeting is for general informational purposes only and is not intended to be a substitute for professional medical advice, diagnosis, or treatment. While the professional you speak with will make every effort to provide accurate and up-to-date information, this session does not establish a provider-patient relationship. Participants are encouraged to consult with their own healthcare providers regarding any specific medical concerns or conditions. Do not disregard or delay seeking medical advice based on information obtained during this session. The organizers and participating professionals are not liable for any actions taken or not taken based on the information provided. Meeting Is Not Guaranteed While we will make every effort to match your inquiry with an appropriate expert attending SciFam, we cannot guarantee it due to time limitations, available timeslots, availablity of an expert, and potentially, the nature of your inquiry. If you are comfortable asking your question during plenary or subtype-specific breakout sessions, please use these forums first. If we are unable to book your requested meeting, you will be notified as soon as possible, but likely no sooner than July 15, 2025. Personally identifying information and the nature of your inquiry provided in this secure form will only be shared with the clinician you are assigned for the 1:1 meeting.

Attendance Policies All attendees must be registered, and must wear their badge at all times while on the conference floors. This is largely to ensure the safety of our young attendees - that no one who doesn't belong to our stakeholder community can gain access to them. So please make it a priority to pick up your conference badge as soon as possible upon arrival, and remember to wear it at all times while attending sessions. The registration desk will be open during all scheduled sessions. Thank you for your cooperation. While we will not be instituting vaccination or mask requirements, please be mindful that our affected community members can be particularly vulnerable to illness. So if you are experiencing any transmittable symptoms, please wear a mask, wash your hands frequently, and keep your distance from other attendees. If, during the course of the conference, or in the two weeks following, you are diagnosed with a communicable disease, please let us know immediately.(https://www.scifam.info/contact) Refund Policy Should something come up, and you are no longer able to attend SciFam, fees paid for registration, Kids Camp, and meals may be refunded until June 30, 2025, upon request (https://www.scifam.info/contact). After this date, fees paid cannot be refunded as we will have already committed to expenses associated with your attendance. If cancelling your attendance, you have the option to convert your paid fees as a donation to Cure CMD, A Foundation Building Strength, or Team Titin, from whom you will receive an appropriate gift acknowledgement. Hotel reservations may be cancelled up to 3 days prior to your scheduled arrival. After this date, the first night will be charged to your credit card by the hotel directly. This includes sponsored attendees.