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.
TOP STORIES
NATIONAL PRESS
This is just a selection of recent articles published in the United States. For a full list of national press coverage, view our media archives below.
interNATIONAL PRESS
This is just a selection of recent articles published across the globe. For a full list of international press coverage, view our media archives below.
AUDIo
documentary
Publications
television
CBS News Colorado (Dec. 11, 2019)
FOX 31 Denver (Dec. 2, 2019)
KBS World TV (Jul. 24, 2019)
FOX 5 DC (Oct. 26, 2018)
recent presentations
Oxford-Harrington Rare Disease Centre Rare Disease Day 2023
Genomics England Discovery Forum 2022
RARE-X Exchange Patient Innovator Forum 2022
Speaking Engagements & Awards
Ultragenyx Rare Bootcamp 2024
Economist Impact 2nd annual Cell and Gene Therapy Summit 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
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
University of Oxford Department of Paediatrics IDRM Seminar 2022
MIT Sloan School of Management Healthcare Finance Seminar 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 End-of-Year Meeting 2021
UCSF-Stanford CERSI-FDA Workshop on Prenatal Somatic Cell Gene Therapies 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
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, "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, "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"
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"
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"
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?"
Fox 5 DC, "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"
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, "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, "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, "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"
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"
The Times, "Drug created for Mila Makovec, only girl with variant of Batten Disease"
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
NETHERLANDS
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"
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
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
SLOVAKIA
The Pravda Daily, "Dievčatku s Battenovou chorobou podali špeciálne vyrobený liek"
ROMANIA
BULGARIA
Mediapool.bg, "Учените са създали лек за един пациент"
RUSSIA
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"
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
THAILAND
INDIA
India Times, " सिर्फ एक मरीज के इस्तेमाल के लिए बनाई गई खास दवा"
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
BRAZIL
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.