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"

• 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, "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, "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"

• 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"

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."

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.