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Home - Selecta Biosciences
Unlocking the Power of Biologics
Home - La Force DMD
Author: Marie-Catherine Du Berger
2nd Gene Therapy for Muscular Disorders Summit | April 2022 | Home
The 2nd Gene Therapy for Muscular Disorders Summit will focus on overcoming the challenges of developing genetic therapies targeting muscular disorders, including Duchenne Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Pompe Disease and X-Linked Myotubular Myopathy.
Jesse's Journey | Duchenne Muscular Dystrophy | Canada
Duchenne Muscular Dystrophy (DMD) is a progressive genetic muscular system disease that affects 1 in every 5,000 boys.
Home - Progena
Progena is a Swiss Duchenne Foundation for patients with Duchenne muscular dystrophy, their families, doctors, therapists.
Home Page 1 - Pietro's Fight
Help Pietro fight Duchenne Muscular Dystrophy (DMD). Join us in learning more about this disease that affects 300,000 children worldwide and donate to help fund the fight for a cure. This is our mission of love and our fight for a cure!
Consortium.AI
Consortium.AI - the joined force to apply the latest advances in AI to discovery of novel small molecules for the Duchenne Muscular Dystrophy (DMD) and other rare orphan diseases.
Neurogene | Developing life-changing Genetic Medicines
Neurogene is focused on developing life-changing genetic medicines for patients & families affected by rare, devastating neurological diseases.
Parent Project Muscular Dystrophy (PPMD) | Fighting to End Duchenne
Parent Project Muscular Dystrophy fights to end Duchenne. We accelerate research, raise our voices to impact policy, demand optimal care for every single family, and strive to ensure access to approved therapies.
Homepage Ipsen - Fondation Ipsen
Rare Diseases Rare but not alone Mission Accelerate rare disease diagnosis There are 7,000 rare diseases affecting 300 million people worldwide. 75% of patients are children. 1 in 2 patients do not have an accurate diagnosis. A quarter of patients wait 4 years to get a diagnosis. Our program brings together world experts to improve … Homepage Ipsen Read More »
Leadiant Biosciences - Research Based Pharmaceautical Company
At Leadiant Biosciences, Inc., our legacy of serving patients is as strong as the future we envision. We developing life-changing therapies for people with rare diseases worldwide.
William E. Proudford Sickle Cell Fund
Our Mission: To support sickle cell awareness, education, state-of-the-art treatment and research, and to bring hope to families affected by this devastating disease.
Kriya Therapeutics - Unleashing the power of gene therapy
Our mission at Kriya Therapeutics is to expand the reach of gene therapy to address highly prevalent diseases affecting millions of patients.
Travere Therapeutics
Travere Therapeutics is a biopharmaceutical company whose mission is to identify, develop and deliver life-changing therapies to people living with rare disease.
Author: Superadmin
Project Sebastian A Rare Disease Foundation Funding Support
We at Project Sebastian gives support, funding and hope to families affected by a child with a rare disease. Provide education, therapies, and counseling.
Alex's Wish | fundraising charity for Duchenne Muscular Dystrophy
Alex’s Wish is a charity set up to eradicate Duchenne Muscular Dystrophy, an aggressive form of Muscular Dystrophy that affects 1 in every 3,500 boys born and results in total paralysis. Established by Emma and Andy Hallam (parents to Alex) in late 2012, Alex's Wish helps raise vital funds to help bring about new treatments and ultimately a cure for this devastating condition.
The French Muscular Dystrophy Association (AFM-Téléthon)
AFM-Téléthon is an association composed of patients and their families who are affected by a genetic, rare, progressive and severely disabling disease
Steve Waugh Foundation | Rare Disease Foundation Australia
Steve Waugh Foundation's mission is to enhance the lives of children and families affected by a rare disease. Grants and fundraising assistance available.
Lung Health Institute | Cellular Therapy for Lung Disease | Breathe Easier™
At the Lung Health Institute, our primary focus is to help patients with chronic lung disease Breathe Easier™. We offer cellular therapy treatments that..
Other sites like catabasis com
Amolyt Pharma - Advancing peptides for the treatment of rare endocrine and metabolic disorders
We are building on our team's established expertise in therapeutic peptides to deliver life-changing treatments to patients suffering from rare endocrine and metabolic diseases.
Author: Jpoly4
Zogenix - Therapeutic Solutions for Rare Diseases
Zogenix is committed to developing and commercializing transformative therapies to improve the lives of patients and their families living with rare diseases.
Comradis
AMICULUM Comradis is a specialist healthcare communications and commercialization consultancy. Our priority is to ensure that patients and families affected by rare diseases and uncommon cancers receive the best possible care and support
Leber Congenital Amaurosis Non-Profit | Hope in Focus
Hope in Focus is a non-profit organization dedicated to providing information and support to those affected by LCA and other rare inherited retinal diseases.
Author: Sofia Sees Hope
RareConnect
A safe, easy to use platform where rare disease patients, families and patient organizations can develop online communities and conversations across continents and languages. RareConnect partners with the world's leading rare disease patient groups to offer global online communities allowing people to connect around issues which affect them while living with a rare disease.
Welcome to Eton Pharmaceuticals
Eton is an innovative pharmaceutical company that searches the world over for meaningful therapies that we can bring to patients living with rare diseases.
ScharlLab - Gastroscience Michael Scharl
Our research aims at improving the quality of life and therpeutic options of our patients affected by gastrointestinal diseases.
Home | Hope for Hearts
Hope for Hearts MISSION Our mission is to help change the life expectancy and the quality of life of children with heart disease and chronic medical conditions by supporting programs that promote an active and healthy lifestyle. INSPIRATION “Life is more than a beating heart. Our next challenge is to make sure that our patients will live at
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Rare2Aware – Bringing hope to people with rare diseases
Rare2Aware is a place to learn about rare diseases and bring together patients, their family, caregivers, healthcare professional and advocates
Principia Biopharma | Tailored Covalency | BTK inhibitors
Principia is bringing transformative therapies to patients with serious immune-mediated diseases by changing the way small molecules are developed.
Rare diseases Luxembourg , we care 4 rare! - ALAN
We aim to improve the quality of life of individuals and families affected by rare diseases in Luxembourg. Discover how we can help you.
Home
The Hope House Memphis mission is to improve the quality of life for ****-affected individuals and their families by providing high quality early childhood education and social services. Hope House will be a catalyst for life-long change in the lives of those affected by **** and poverty.
Home | Arlington Pediatric Therapy
The mission of Arlington Pediatric Therapy Management Services (APTMS) is to provide quality therapy for children on an outpatient basis. Our primary focus is
Doctors On Duty - Family Doctor - Mississauga, Ontario
At our family medicine clinic in Mississauga, we focus on providing patients with a comprehensive approach to primary care.
home - FunctionalGenomics
WELCOME TO PAWEL LISOWSKI RESEARCH WEBSITE SOLVING MEDICAL MYSTERIES THROUGH GENOME ENGINEERING TECHNOLOGY Decoding of undiagnosed and rare diseasesHealthcare providers are unable to discover the cause for hundreds mysterious diseases every year. Our research study aims to provide answers for patients and families affected by these undiagnosed conditions. We focus on decoding of unknown and...
Whittemore Peterson Institute – Medical Research Institute
Our Story Whittemore Peterson Institute (WPI) sprang from hope and determination to change the course of a spectrum of disabling diseases that continue to deeply affect our founders’ family and thousands of other families within the state of Nevada and around the world. Our founders asked a
Author: NVCBR
Infectious Disease Consultants of Georgia | 6 Atlanta Locations
Physicians Focusing On Care Five out-patient locations in the Greater Atlanta area Make an Appointment About Infectious Disease Consultants At Infectious Disease Consultants our primary goal is to focus on patient care.Infectious Disease Consultants is operated in six out-patient locations in the greater Atlanta area. Our many physicians are dual board certified in infectious disease and internal
Forge Biologics - A GMP/ cGMP Compliant CDMO
Forge is a gene therapy development company, focused on encouraging hope and bringing life changing gene therapies from idea to reality.
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Pierce Family Dentistry is a small family-run dentist office in Mt. Pleasant, South Carolina. Our primary focus is on our patients. Your smile is important!
Cure Rare Disease
Cure Rare Disease™ is developing customized therapeutics for those who have been diagnosed with rare, genetic diseases that have no treatment or cures. Through partnerships and collaboration, our world-renowned researchers build life-saving therapeutics that are custom to the patient they are made to treat.
What else catabasis.com alternative websites
VectivBio | Transforming Lives
Life-Transforming Medicines for Patients Living with Severe Rare Diseases We are a global, clinical-stage biotechnology company focused on the discovery, development and commercialization of innovative treatments for severe rare conditions with high unmet medical need. Learn More About Us Our Focus We use a patient-centric approach to identify and develop transformative medicines for the treatment […]
Homepage | chILD Foundation
We are a 501(c)3 tax-exempt organization that provides support, education, and hope for a cure to all families with children battling these life-threatening diseases. What are chILD disorders? Children’s Interstitial Lung Disease (chILD) is a group of rare pediatric lung diseases. There are more than 30 disorders included under the chILD umbrella. The mission of […]
AllCare Family Medicine & Urgent Care | AllCare Family Medicine & Urgent Care
AllCare is an Innovative Primary Care Group Combining Family Medicine, Urgent Care & Telemedicine. Our Mission: "Happy Doctors, Healthy Patients"
Rare Genetic Diseases | Ultragenyx
Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.
Philippine Society for Orphan Disorders, Inc. – A pathway to support, resources and hope
LEARN MORE ABOUT NRDW 2021 OUR MISSION Our mission is to uphold the primary concerns and welfare of individuals with rare disorders by directly addressing, supporting and protecting their health and general well being ABOUT PSOD identified genetic origins Affect children
Author: Webmaster
Hospice of Southern Maine :: Compassion, Care & Comfort
At Hospice of Southern Maine, our mission is to provide compassion, care and comfort through end of life for patients with life-limiting illnesses, and support for their families.
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515 Therapy and Consulting offers family and individual therapy, conciliation, and child specialists. Our therapy services can help with a number of life's stresses and conflicts that can affect anyone.
Home - LGMD Awareness Foundation
ABOUT US LGMD Awareness Foundation, Inc. is a 501(c)(3) non-profit advocacy organization dedicated to globally raising awareness of the rare neuromuscular conditions known as limb-girdle muscular dystrophy (LGMD). In collaboration with other LGMD foundations, our focus is to provide curated educational information and resources for the LGMD community and public. By increasing
Author: Dooley
Home
ABOUT US LGMD Awareness Foundation, Inc. is a 501(c)(3) non-profit advocacy organization dedicated to globally raising awareness of the rare neuromuscular conditions known as limb-girdle muscular dystrophy (LGMD). In collaboration with other LGMD foundations, our focus is to provide curated educational information and resources for the LGMD community and public. By increasing
Author: Dooley
Home 2021
STREAMING HOPE + THE POWER OF MUSIC A lot has changed in 2020, but our mission of bringing hope, connection and the power of music to those in need has not.
Author: Melodic
Foundation Fighting Blindness — Foundation Fighting Blindness
The urgent mission of the Foundation Fighting Blindness is to drive the research that will provide preventions, treatments and cures for people affected by retinitis pigmentosa, age-related macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases. The Foundation is a beacon of hope for those affected by these blinding diseases. Join the fight and help us accelerate our mission.
Home - NUBPL.org
Our mission is to raise awareness and fund research towards the development of life-saving treatments and a cure for NUBPL, a mitochondrial complex 1 deficiency disorder. NUBPL is a progressive neurodegenerative disease that affects children. There are zero FDA approved treatments for this disease. Patients with NUBPL present clinical symptoms between the ages of 3-24 months, including
Author: Nubpl
Home | AFSP
Learn about suicide, how you can help prevent it, and resources for those affected, from the American Foundation for Suicide Prevention. Our mission: save lives and bring hope to those affected by suicide
Johannesburg Parent & Child Counselling Centre (JPCCC)
Our mission is the holistic development of individuals, families & communities through counselling & therapy, assessments, life skills, training & development
DJ's Hope 4 Hearts
DJ's Hope 4 Hearts' mission is to provide financial support to families that struggle during challenging times, or during the hardships due to a sick child. Our primary focus is dedicated to the healing hearts of children so that he or she can grow in love, peace, and happiness. We also seek to become an educational resource for children and their families on the symptoms of cardiomyopathy and other deadly heart diseases via this website.
Home | The Seany Foundation
The Seany Foundation brings hope and happiness to kids affected by cancer. Our diverse programs help patients, siblings and parents reclaim the joy cancer often
LifePetitions - Petitions for a Culture of Life
LifePetitions is a petition platform exclusively serving the pro-life and pro-family communities. Our goal is to bring about concrete change and create a Culture of Life.
Home
Giving Men Hope A Future, and a Purpose Our program is based on the principle that only Christ can truly change a life. Our primary goal is to help
Author: - Ben H
mission - Facio Therapies
Change the narrative. We are a biology-oriented company founded by people living with facioscapulohumeral dystrophy (FSHD), a progressive muscle disease with a very high unmet medical need. There is currently no approved therapy available – so we are laser-focused on developing one. To maximize our chance of success, we do not bet on a single... read more
Pulmonologist Fourways Sandton | Dr Muhammad Chohan & Dr Bronwyn Schär
Pulmonologist in Fourways, Sandton, Johannesburg. At Lungs for Life our primary focus is on maintaining lung health and helping our patients breathe easier. We assess and manage adult patients with a wide variety of acute and chronic respiratory problems.
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Home | East Memphis Direct Primary Care
East Memphis Direct Primary Care brings back the patient experience of personal healthcare delivered by a trusted/personal healthcare provider, cutting out the insurance middleman so we can focus on taking care of you and your family.
Advancing health outcomes through technology, data science, and a team of experts across clinical research, care, and devices | Verily Life
Verily is focused on using technology to better understand health, as well as prevent, detect, and manage disease. Our mission is to bring together technology and life sciences to uncover new truths about health and disease.
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Verily is focused on using technology to better understand health, as well as prevent, detect, and manage disease. Our mission is to bring together technology and life sciences to uncover new truths about health and disease.
Author: Вызвать эвакуатор автомбилей в Москве и Московской области недорого
HOME - Trappe Family Practice
Trappe Family Practice is a primary care practice offering convenient access to compassionate care and advanced medicine, serving patients throughout every stage of life – from infancy to adulthood. Patients seen by our primary and specialty care physicians have access to the entire Einstein Healthcare Network.
Hospice care, palliative care & grief support in Wisconsin - Unity Hospice
Our Mission At Unity, caring is at the heart of our patient-focused mission: To bring the best end-of-life experience to our communities by delivering unwavering strength, compassion and support. Why Choose Unity? Our Services As the first and most trusted hospice
Author: Unityadm
Younique Therapies
At Younique, we are able to offer patients access to cutting-edge adipose stem cell therapies for degenerative diseases, cosmetic enhancements and athletic procedures. Through our stem cell studies, Younique hopes to provide patients with options that may change the course of their lives and benefit them in ways they never imagined possible.
Author: Stem Cell Therapy Studies
Audentes Homepage
Developing TherapiesTo Transform Lives Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Innovative Therapies Our investigational therapies target rare diseases through three modalities: gene replacement, exon skipping gene therapy and vectorized RNA knockdown.
Author: Wayne Stedman
Home - Gyroscope
Vision for LifeDEVELOPING ONE-TIME GENE THERAPIES FOR EYE DISEASES Vision for LifeDEVELOPING ONE-TIME GENE THERAPIES FOR EYE DISEASES 3 Welcome to Gyroscope We are discovering and developing innovative medicines to preserve people’s sight and fight the devastating impact of blindness around the world. Our ultimate goal for our patients is – Vision For Life. […]
Author: GyroscopeTX
HOME - VPR Patient Outreach Program
Patient Speakers Patient-To-Patient Programs People Reached! Bring life to your brand with real patients and real stories. Let our experienced team and resources guide you in taking your brand to the next level. Patients recruited and trained to share their powerful journeys and disease education.
Author: Nick
Home - Careity Foundation
OUR MISSION Careity exists to provide direct care that saves lives, gives hope, eases pain, and allows cancer patients and families to focus on healing. Careity Foundation is a Texas 501(c)3 Read More of direct patient services provided in 2020 patient visits in 2020 chemotherapy grants provided ...
Author: Mike Branch
Inspiration Research Limited
Caring For Life Our goal is to educate patients to better manage their diseases and contribute to the advancement of novel therapies. OFFICE HOURS Monday - Friday Appointment only Saturday Appointment only
Author: Admin
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At Paragon Sports Medicine, we stay on top of the latest research and science behind all the regenerative medicine techniques to help our active patients recover faster. Without surgery, it is possible to utilize the body’s own stem cells to manage and recover from numerous musculoskeletal injuries.
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Digimore Electronics Co., Ltd
With 20 years experience engineers of PC peripherals, Digimore Electronics provides ODM services with different modules including Bluetooth BLE modules, biometric/ fingerprint/ Finger Vein modules, keyboard modules and touch pad module for ****, and etc.
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Home | Video Stories
With a love for telling meaningful stories. We want to inspire people all around the world with meaningful stories. Stories that need to be told, because they simply matter. In this visual culture of today, video is a great tool to communicate and to reach out to anyone who wants to be inspired by your story.
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Somos un grupo de profesionales que tiene como objetivo mejorar la calidad de vida de las personas mediante la actividad física, logrando introducir la vida saludable dentro del ambiente laboral de cada una.
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