MEDIA

Mila's story has gained national and international attention as she became the first person in the world to receive a drug tailored just to her. Together with Dr. Timothy Yu and Boston Children’s Hospital, our foundation has forged a new path of individualized medicines for rare disease in children.

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NATIONAL PRESS

interNATIONAL PRESS

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Publications

recent presentations

 Speaking Engagements & awards

• Personalized Medicine Conference 2024

• N=1 Collaborative Annual Meeting 2024

• Oxford-Harrington Rare Disease Centre Symposium 2024

• ICMRA ‘Rare’ Symposium 2024

• American College of Clinical Pharmacology (ACCP) Annual Meeting 2024

• Lilly Seaport Innovation Center Grand Opening Celebration 2024

• Project 8p Family And Science Conference 2024

• Ultragenyx Rare Bootcamp 2024

• World Orphan Drug Congress USA 2024

• Economist Impact 2nd Annual Cell and Gene Therapy Summit 2024

• STAT Breakthrough Summit East 2024

• Mayo Clinic CIM Grand Rounds Rare Disease Day Special Webinar 2024

• CIRM Rare Diseases Workshop 2023

• 19th Annual Meeting of the Oligonucleotide Therapeutics Society 2023

• Genomics England Research Summit 2023

• CCDS Virtual Conference 2023

• Ultragenyx’s Gene Therapy Facility Room Dedication 2023

• ASGCT 26th Annual Meeting 2023

• UCSF Prenatal Somatic Cell Gene Therapy & Gene Editing (PSCGT/E) – Ethical Considerations 2023

• RE(ACT) Congress & IRDiRC Conference 2023

• Harvard Medical School Boehringer Ingelheim Executive Education Webinar 2023

• Oxford-Harrington Rare Disease Centre Rare Disease Day Webinar 2023

• Danaher Leadership Conference 2023

• TREAT-NMD Conference 2022

• University of Oxford Department of Paediatrics IDRM Seminar 2022

• Genomics England Discovery Forum 2022

• MIT Sloan School of Management Healthcare Finance Seminar 2022

• RARE-X Exchange Patient Innovator Forum 2022

• Global Genes RARE Patient Advocacy Summit 2022

• Ultragenyx Leadership Summit 2022

• RNA Therapeutics Symposium 2022

• ASGCT Annual Meeting 2022

• ASCPT Annual Meeting 2022

• N=1 Collaborative Annual Meeting 2021

• UCSF-Stanford CERSI-FDA Workshop on Prenatal Somatic Cell Gene Therapies 2021

• TEDxLungarnoMediceo 2021

• BDSRA Annual Family Conference 2021

• NIH Gene-Targeted Therapies Meeting 2021

• CZI n-of-1 Meeting Series 2021

• NYU Langone Health Pediatric Gene Therapy & Medical Ethics Working Group 2021

• BIO: Women’s History Month 2021 (28 pioneering women in science and biotechnology to know)

• DISORDER: The Rare Disease Film Festival Film Selection 2020

• NORD Rare Impact Award Honoree 2020

• Colorado BioScience Association Briefing 2020

• NORD RareLaunch® Forming a Foundation Workshop 2020

• CZI Neurodegeneration Challenge Network Meeting 2020

• Harvard Medical School Precision Medicine Conference 2020

• NIH Rare Disease Day Panel on Personalized Medicine 2020

• FDA Rare Disease Day Panel on Individualized Therapies 2020

• Children’s Hospital Colorado’s NSI Burns Night 2020

• The Precision Medicine World Conference (PMWC) 2020

• FDA Meeting: Promoting Effective Drug Development Programs 2019

• Stanford Medicine Pediatric Grand Rounds 2019

• GoldLab Symposium 2019

• FDA’s Critical Path Innovation Meeting 2019

• MIT’s Broad Institute Rare Disease Day 2019

• Foundation for Angelman Syndrome Therapeutics Gala 2018

• Boston Children’s Hospital’s Manton Center for Orphan Disease Research 10th Anniversary 2018

• Undiagnosed Disease Network UDN Steering Committee 2018

• American Society of Human Genetics Conference 2018

 MEDIA ARCHIVEs

United StateS

• STAT, "Getting CRISPR treatments to patients remains a major challenge. Could lab-developed tests help?"

• Endpoints News, "'This is not a science problem anymore': Paths emerge for scaling up rare disease medicine"

• STAT, "A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments"

• Nature Biotechnology, "Personalized medicine is having its day"

• The New York Times, "They Created a Drug for Susannah. What About Millions of Other Patients?"

• STAT, "A mother, shaped by tragedy, embarks on a mission to advance custom medicines"

• 5280 Magazine, "The Colorado Parents Fighting the World's Rarest Diseases"

• Good Day BIO, "Women’s History Month: 28 pioneering women in science and biotechnology to know"

• Batten Disease News, "FDA Issues Guidance on Developing Rare Disease Treatments"

• Boston Children’s Discoveries, "Made-to-order therapies get a boost with new FDA guidelines"

• CEO Magazine, "Four lessons Zuckerberg and Chan have learned from philanthropy"

• Chan Zuckerberg Initiative, "Our 2020 Annual Letter"

• Charles River, "Corporate Citizen Report 2020"

• National Organization for Rare Disorders, "NORD Introduces This Year’s Rare Disease-Fighting Super Group: The 2020 Rare Impact Award Honorees!"

• The New York Times, "What We Learned in 2019: Health and Medicine"

• Times-Call, "Boulder County family raises money for research on personalized medicines at Children’s Hospital Colorado"

• CBS Denver (Dec. 11, 2019), "Mila Makovec Now First Ever To Receive FDA Approved Drug For 1 Person"

• STAT Plus, "12 burning questions for Stephen Hahn, Trump’s pick to lead FDA"

• Fox Denver 31 (Dec. 2, 2019), "Boulder girl is first person to receive FDA-approved drug created for one patient"

• Chemical & Engineering News, "C&EN’s Year in Pharma 2019"

• The Colorado Sun, "This miracle drug was designed and manufactured for just one person — a 9-year-old Boulder girl"

• Fox Denver 31, "FDA approves one-of-a-kind drug for 9-year-old in Boulder"

• The Denver Channel, "Drug designed and manufactured for 9-year-old Boulder girl"

• The Sacramento Bee, "Drug designed and manufactured for 9-year-old Boulder girl"

• San Francisco Chronicle, "Drug designed and manufactured for 9-year-old Boulder girl"

• My San Antonio, "Drug designed and manufactured for 9-year-old Boulder girl"

• My Journal Courier, "Drug designed and manufactured for 9-year-old Boulder girl"

• Times Union, "Drug designed and manufactured for 9-year-old Boulder girl"

• The Hour, "Drug designed and manufactured for 9-year-old Boulder girl"

• Sky Statement, "Local News Drug designed and manufactured for 9-year-old Boulder girl The Associated Press 10"

• Global Genes, "The Mother of Invention Strikes Again"

• The New England Journal of Medicine, "Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease"

• The New England Journal of Medicine, "Drug Regulation in the Era of Individualized Therapies"

• NIH Director’s Blog, "One Little Girl's Story Highlights the Promise of Precision Medicine"

• Science Magazine, "Drug tailored to one girl with brain disease paves way for similar customized treatments"

• The New York Times, "Scientists Designed a Drug for Just One Patient. Her Name Is Mila."

• MIT Technology Review, "This girl’s dramatic story shows hyper-personalized medicine is possible—and costly"

• STAT, "After a bespoke therapy rescues a young girl, the FDA considers advance of individualized treatments"

• Northwestern University, "Northwestern Helps Develop Custom Drug"

• MedPage Today, "Mila's N-of-1 Trial Detailed in NEJM"

• 90.9 WBUR Boston, "A Drug Was Made For Just One Child, Raising Hopes About Future Of Tailored Medicine"

• Batten Disease News, "Customized Oligonucleotides Treat Seizures in Child with CLN7 Disease in Record Time"

• Eureka, "Medicine for Mila: How physicians and scientists teamed up for one little girl born with a deadly form of Batten disease"

• Fox News, "Girl facing fatal brain disease gets tailor-made drug"

• Advisory Board, "Meet 'Milasen': The first-ever drug designed for just one patient"

• Live Science, "Girl Diagnosed with Fatal Brain Disease Gets a Tailor-Made Drug within a Year"

• Pink Sheet, "One Patient, Many Questions: Milasen ‘N of 1’ Trial Provides Early Template For Individualized Genomic Medicine"

• EurekAlert!, "New customized drug treatment bypasses a single child's unique mutation within a year of diagnosis"

• CTV News, "Eight-year-old girl gets personalized drug for rare disease after parents raise millions"

• Science Codex, "New customized drug treatment bypasses a single child's unique mutation within a year of diagnosis"

• Health Gazette, "Scientists designed a drug for just one patient. Her name is Mila."

• Clinical OMICs, "BCH N of 1 Trial Yields Approved Therapy for Single Rare Disease Patient"

• Guru Online News, "Unique drug for a girl with deadly brain disease"

• Nature Medicine, "Antisense therapies pose a regulatory conundrum"

• MedPage Today, "Desperate Families Pursue 'N-of-1' Trials for Ultra-Rare Diseases"

• Boston Children’s Hospital Trust, Spirit of Giving, The Dare Issue, "In Rare Form"

• NeurologyToday, "An Antisense Oligonucleotide Therapy Looks Promising for a Rare Form of Batten Disease"

• Left Hand Valley Courier, "Mila’s Miracle featured at CU’s GoldLab Symposium"

• Chemical & Engineering News, "Have antisense oligonucleotides hit their stride?"

• BioQuick News, "Patient-Customized Anti-Sense Therapy Possibly Successful for Young Girl with MFSD8/CLN7 Batten Disease, an Ultra-Rare and Generally Fatal Genetic Disease"

• Fox 5 DC (Oct. 26, 2018), "New customized drug is giving young girl suffering from rare brain disease new hope for cure"

• The Boston Globe, "Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease"

• STAT, "Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease"

• Science Magazine, “A tailormade drug developed in record time may save girl from fatal brain disease”

• MIT Technology Review, “Gene treatment made for a single child cuts a hyper-personal path for precision medicine”

• Inquisitr, “Record Drug Development May Save Girl From Fatal Brain Disease”

• Patient Worthy, "A Girl From Colorado is Receiving An Experimental Treatment for Her Batten Disease"

• Left Hand Valley Courier, "Mila starts leading-edge treatment"

• Times-Call, "Boulder County girl undergoing experimental treatment for Batten disease"

• Boulder Patch, "Batten Disease: Gene Therapy Offers Hope for Boulder Girl"

• Boulder Lifestyle, "Finding a Miracle"

• Forbes, "Rapper B.o.B Just Started A GoFundMe To Prove The Earth Is Flat"

• Times-Call, "Boulder County family down to wire in fight against rare disease"

• Buzz.ie, "Family must raise $1M to save their 6-year-old daughter's life"

• Fox 31, "Boulder family races to raise $1 million for clinical trial for daughter’s deadly disease"

• New York Post, "Family races to save 6-year-old with fatal genetic disease"

• Fox 31, "Family racing to raise funds for daughter facing incurable genetic disease"

• Today Show, "Parents of little girl with rare, deadly disease race time to save her"

• Climbing, "Mila Makovec: A Little Climber in Need of Our Help"

• Times-Call, "Boulder County family is funding race against deadly Batten disease"

• Fox 31, "Governor’s wife joins fight to help family raise $4 million for clinical trial"

• Fox 31, "Teachers school each other to help families’ wishes come true"

• Fox 31 (Feb. 4, 2017), "Charity steps up to help family raise $4 million to find cure for daughter’s deadly disease"

• The Denver Post, "Family’s struggle with Batten disease mirrors the journey of others fighting rare diseases"

• Fox 31 (Jan. 16, 2017), "Family trying to raise millions to fight daughter’s rare, deadly disease"

• Daily Camera Longmont News, "Boulder County family desperate to raise money to combat rare Batten disease"

• Daily Camera, "Photos: Boulder County Girl Diagnosed with Batten Disease"

• ABC Denver 7 (Jan. 12, 2017), "Longmont girl battles rare disease with no cure in sight"

CANADA

• Daily Stock Dish, "Eight-year-old girl gets personalized drug for rare disease after parents raise millions"

ENGLAND

• BBC Radio 4, "Mila’s Legacy: One mother’s efforts to save her child could start a revolution in personalised medicine."

• The Telegraph, "Mila’s legacy: The little girl who could change how medicines are made in Britain"

• Genomics England, "Pilot launched to support children with rare conditions to access personalised therapies"

• The Economist, "The world’s first pathway for individually designed drugs"

• BBC News, "The quest for the era of personalised medicine"

• Wired UK, "A dying child, a mother’s love and the drug that changed medicine"

• BBC News, "'Man on the Moon' moment - the year's big breakthroughs"

• The Economist, "The World in 2020"

• BBC News, "Batten disease girl given custom-made drug"

• Daily Mail, "'She's still herself': Girl, 8, left blind and mute by an incurable rare brain disease is the first patient to get a drug tailor-made - and named - for her in historic step for personal medicine"

• The Times, "Drug created for Mila Makovec, only girl with variant of Batten Disease"

• Daily Mail, “World first as experimental drug appears to REVERSE symptoms in girl, 7, with incurable disease that causes paralysis, blindness, dementia and early death”

• Daily Mail, "Tragedy of Girl, 6, Who Suffers from Rare Disease that Will Rob Her of Speech, Mobility and Eyesight — and Could Kill Her Before She's a Teen"

IRELAND

• The Irish Times, "The drug created to treat just one patient"

SWEDEN

• Life Science Sweden, "Första läkemedlet för bara en patient"

GERMANY

• Süddeutsche Zeitung, "Ein Medikament nur für Mila"

• Deutsches Ärzteblatt, "Forscher stellen Wirkstoff für eine einzige Patientin mit Batten-Syndrom her"

BELGIUM

• DeMorgen, "Artsen ontwikkelen genetisch medicijn dat maar voor één meisje werkt (en het kost 3 miljoen dollar)"

NETHERLANDS

• de Volkskrant, "Amerikaanse artsen maken genetisch medicijn voor één meisje"

FRANCE

• Le Monde, "Un traitement ultra-personnalisé pour une fillette souffrant d’une maladie génétique rare"

ITALY

• Le Iene, "Malattie rare: bambini lasciati soli senza una cura"

• la Repubblica, "Per la prima volta messo a punto farmaco per un singolo paziente"

• Corriere Della Sera, "Farmaco creato per una sola paziente Mila, 8 anni, ha la malattia di Batten"

• FanPage.it, "Creato farmaco rivoluzionario per trattare un singolo paziente con una rarissima malattia genetica"

• Quotidiano.net, "Creato farmaco per una sola paziente: cura per una bimba con la malattia di Batten"

• L’HuffPost, "Scienziati creano un farmaco per una sola paziente: Mila, 8 anni, ha una sindrome rarissima"

• Open, "Nuovo farmaco sperimentato su Mila, la bimba affetta dal morbo di Batten. Ma attenzione a non dare false speranze"

• Il Post, "Il primo farmaco creato per un solo paziente"

• Nurse Times, "Boston, creato un farmaco personalizzato per curare la piccola Mila"

• Il Tempo, "Scienziati Usa creano il primo farmaco per una sola persona al mondo"

• Sputnik Italia, "USA, equipe di scienziati brevetta cura per una sola paziente"

• News Mondo, "Ideato un farmaco per una sola persona"

• ANSA Italia, "Per la prima volta un farmaco creato per un singolo paziente"

• AGI - Agenzia Giornalistica Italia, "Una bambina di 6 anni è stata salvata con un farmaco fatto apposta per lei"

• AssoCareNews.it, "Farmaci orfani: creato farmaco per una unica paziente."

• Focus, "Un farmaco che porta il tuo nome: l'era della medicina ultrapersonalizzata"

• Quotidiano Sanità, "Malattie rare. 'Milasen': negli Usa il primo farmaco creato apposta per un solo paziente"

• Adnkronos, "Il genetista, 'farmaco su misura solo un primo successo'"

• Bigodino.it, "Mila, colpita da una rara malattia genetica neurodegenerativa"

• Socialfarma, "Creato il primo farmaco per un singolo paziente"

SWITZERLAND

• Tages-Anzeiger, "Ein Medikament nur für Mila"

SPAIN

• El País Espana, "Creado por primera vez un fármaco para un único paciente: una niña con una enfermedad letal"

• ABC España, "Milasen: el primer fármaco ‘personalizado’ que salva a la niña con una enfermedad cerebral mortal"

• 20 Minutos, "Crean un fármaco para un único paciente: una niña con una enfermedad mortal"

• Televisió de Catalunya 324, "Creen un fàrmac per a un sol pacient: una nena amb una malaltia mortal"

PORTUGAL

• Rynekaptek.pl, "Terapie tworzone tylko dla jednej osoby. Jakie mogą nieść korzyści innym?"

GREECE

• Athens Voice, "Εξατομικευμένο φάρμακο για μία και μόνο ασθενή, δίνει ελπίδα σε πολλούς"

• Nooz, "Ανοίγει ο δρόμος της εξατομικευμένης ιατρικής- Το παράδειγμα "Μίλα"

• Parallaxi Mag, "Επιστήμονες δημιούργησαν νέο φάρμακο σώζοντας ένα κοριτσάκι με θανατηφόρα νόσο"

• Protagon, "Βοστώνη: Εσωσαν οκτάχρονη με νέο φάρμακο που δημιούργησαν ειδικά για αυτήν"

• iPop, "#SavingMila: Το φάρμακο που δημιουργήθηκε αποκλειστικά για να σωθεί ένα 8χρονο κορίτσι"

• Offsite CNN Greece, "#SavingMila:Δημιούργησαν εξατομικευμένο φάρμακο και έσωσαν 8χρονη"

• SKAI.gr, "Εξατομικευμένη Ιατρική: Έσωσαν τη ζωή 8χρονης με φάρμακο ειδικά για εκείνη"

• LiFO, "Έφτιαξαν εξατομικευμένο φάρμακο για 8χρονη με θανατηφόρα εγκεφαλική νόσο"

CROATIA

• JutarnjiLife, "DOKTORI OSMISLILI NOVI LIJEK SAMO ZA NJU Priča o teško oboljeloj Mili Makovec: 'Pomislili smo da joj možemo pomoći i napravili smo lijek Milasen...'"

• Express, "Ovo je Mila i jedina na svijetu ima svoj vlastiti lijek - milasen"

SLOVAKIA

• The Pravda Daily, "Dievčatku s Battenovou chorobou podali špeciálne vyrobený liek"

ROMANIA

• EVZ.ro, "Alertă medicală! S-a inventat medicamentul pentru un singur pacient"

• EVZ.ro, "Premieră mondială! S-a creat un medicament pentru o singură pacientă. Cazul a impresionat lumea medicinei"

BULGARIA

• Mediapool.bg, "Учените са създали лек за един пациент"

RUSSIA

• Vesti.Ru, "Milasen: впервые в мире создано лекарство для лечения единственной пациентки"

CHINA

• WuXi PharmaTech, "这些只能治疗一名患者的疗法，或将带来个体化医疗的新时代"

• Caixin, " 解药｜世界首例个人定制药物是如何问世的"

• ScienceNet.cn, "定制药物治疗脑病见成效"

• Xinhua Net, "First DNA personalized drug used to treat rare disease in U.S."

JAPAN

• Nikkei Biotechnology & Business, "1人の少女に創製・投与された核酸医薬、希少疾患のビジネスモデル模索する母親"

South Korea

• KBS World TV (Jul. 24, 2019), "암 환자도 맞춤형 치료가 가능할까- (20.03.18) 스페셜 암과의 전쟁-면역항암제와 개인맞춤 항암치료"

TAIWAN

• HeHo Health, "[全球第一] 讓9歲的女孩有活下去的權力 科學家量身打造新藥「個性化治療」揭開里程碑"

VIETNAM

• GenK, "Một cô bé mắc bệnh hiểm nghèo, các nhà khoa học đã điều chế riêng một loại thuốc chữa cho cô ấy: Tại sao đó lại là câu chuyện buồn nhiều hơn vui?"

• VnExpress, "Loại thuốc được bào chế riêng cho một bệnh nhân"

THAILAND

• BearTai, "Malisen ยาที่ถูกผลิตมาเพื่อเด็กคนเดียว! หรือนี่จะเป็นอนาคตใหม่ทางการแพทย์เฉพาะบุคคล"

INDIA

• India Times, " सिर्फ एक मरीज के इस्तेमाल के लिए बनाई गई खास दवा"

• The Alayaran, "US doctors customise drug to treat girl with rare genetic disease"

UNITED ARAB EMIRATES

• Gulf News, "A drug invented for just one patient"

SOUTH AFRICA

• Independent Online, "Tech News: Hyper-personalised medicine"

MEXICO

• Agencia ID (Comunicado de prensa), "Mila, el ejemplo de que la medicina personalizada es cara pero real"

ARGENTINA

• El Debate, "Científicos diseñaron un medicamento para una sola paciente: se llama Mila"

BRAZIL

• Portal R7, "Cientistas projetam medicamento para apenas uma paciente"

• Yahoo Notícias,"Como o amor de uma mãe resultou em um dos maiores feitos da medicina"

• Mamãe Prática, "O REMÉDIO DE MILA: COMO O AMOR DE UMA MÃE RESULTOU EM UM DOS MAIORES FEITOS DA MEDICINA"

For media inquiries, contact Julia Vitarello. 

WE NEED YOUR HELP NOW

Mila was given an unprecedented second chance at life through the first-ever truly personal medicine, Milasen. Now we need your help to pave this path for many more children like Mila across hundreds of rare diseases.

Mila's Miracle Foundation, Inc. is a 501(c)(3) nonprofit organization. All donations are tax-deductible.