Osteochondrodysplasia

Tyra Biosciences Announces Appointments of Susan Moran, M.D., M.S.C.E. and S. Michael Rothenberg, M.D., Ph.D. to its Board of Directors

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화요일, 5월 7, 2024
Stanford University, University, FGFR, Disorder, Fibroblast growth factor receptor, Hospital, NDA, QED, Achondroplasia, Hematology, Bile duct, Drug, Research and development, MAA, Yale University, RET, Growth, Duke University, Biotechnology, Therapy, Faculty, Osteochondrodysplasia, Harvard Medical School, Multiple sclerosis, Biology, Drug development, Drug discovery, Liver cancer, Bristol Myers Squibb, Massachusetts General Hospital, Puma Biotechnology, Pharmaceutical industry

and S. Michael Rothenberg, M.D., Ph.D. as independent directors, and the resignation of Isan Chen, M.D.

Key Points: 
  • and S. Michael Rothenberg, M.D., Ph.D. as independent directors, and the resignation of Isan Chen, M.D.
  • "Susan and Michael's impressive careers will provide TYRA with valuable guidance as we advance our potential next-generation precision candidates toward people in need.
  • In connection with these appointments, Dr. Chen, who has served as a director since June 2020, has resigned from the TYRA Board.
  • "On behalf of the Board, I want to thank Isan for his many contributions to TYRA.

Treace Announces First Annual National Bunion Day

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화요일, 4월 16, 2024
Education, Social media, Radar, Foot, 3D, Bunion, Patient, Osteochondrodysplasia, DPM, Quality of life

National Bunion Day is an opportunity to help more people learn about the identification and treatment of this common bone deformity in the foot, which often includes debilitating pain for millions of adults in the U.S.

Key Points: 
  • National Bunion Day is an opportunity to help more people learn about the identification and treatment of this common bone deformity in the foot, which often includes debilitating pain for millions of adults in the U.S.
  • Approximately one in four U.S. adults alone develop the bunion deformity in their lifetime.1
    As the leader in 3D bunion correction, Treace is uniquely positioned to drive greater patient education and awareness of the Lapiplasty® procedure, the #1 most commonly used 3D bunion correction procedure chosen by U.S. surgeons.2 As part of National Bunion Day , Treace is sponsoring a broad-based awareness and information campaign to address misconceptions and stigma associated with bunions.
  • April also marks National Foot Health Awareness Month, an opportunity to set a strong foundation of bunion education and awareness.
  • Updates on activities planned for National Bunion Day are available on the official National Bunion Day webpage at www.lapiplasty.com/NationalBunionDay .

Paley Institute's Global Expansion: Dr. Dror Paley Introduces Instituto Paley LatinoAmerica with Dr. León Mora and CORA Group in Colombia

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수요일, 4월 24, 2024
DSM-IV codes, Partnership, Neurosurgery, PALM, Congenital limb deformities, Osteochondrodysplasia, Patient, Health, Quality of life, Nursing

WEST PALM BEACH, Fla., April 24, 2024 /PRNewswire-PRWeb/ -- Renowned orthopedic surgeon Dr. Dror Paley, in partnership with Dr. León Mora and the Cora Group Excellence Center for Orthopedic Reconstructive and Limb Lengthening Surgery, is proud to announce the incorporation of Instituto Paley LatinoAmerica, a new cutting-edge facility dedicated to advanced orthopedic care. This marks a significant expansion of Dr. Paley's practice into Latin America, reflecting his ongoing commitment to providing world-class orthopedic and neurosurgical services globally.

Key Points: 
  • Instituto Paley LatinoAmerica has officially launched in Medellín, Colombia, in collaboration with Dr. León Mora and the CORA group.
  • Instituto Paley LatinoAmerica will begin welcoming patients on July 19, 2024, in Medellín, Colombia.
  • With over 25,000 successful limb-lengthening and reconstruction procedures to his name, Dr. Paley is a global authority in orthopedic surgery.
  • For more information about Instituto Paley LatinoAmerica, or to schedule an appointment, email [email protected] .

BridgeBio Pharma and Kyowa Kirin Announce Partnership with an Upfront Payment of $100 Million for an Exclusive License on Infigratinib in Skeletal Dysplasias in Japan

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수요일, 2월 7, 2024
Partnership, PALO, License, USD, Osteochondrodysplasia, Child, Safety, MBA, FGFR3, Achondroplasia, Therapy, Kyowa Kirin, Hypochondroplasia, Pharmaceutical industry

In exchange, BridgeBio will receive an upfront payment of USD 100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.

Key Points: 
  • In exchange, BridgeBio will receive an upfront payment of USD 100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.
  • Infigratinib is an oral small molecule designed to inhibit FGFR3 and thus target FGFR3-driven skeletal dysplasias at their source, including achondroplasia and hypochondroplasia.
  • “By partnering with Kyowa Kirin, we hope to significantly accelerate the development of infigratinib to potentially provide options for children with achondroplasia, hypochondroplasia, and eventually skeletal dysplasias in Japan.
  • Based on the results from the latest clinical trials, we believe BridgeBio’s infigratinib has high potential for treating achondroplasia.

Er-Kim Announces Exclusive Distribution Agreement with Ascendis Pharma A/S to Commercialize 3 Endocrinology Disease Treatments Across Central & Eastern Europe and Turkey

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월요일, 1월 8, 2024
Achondroplasia, CFO, ACH, European, Osteochondrodysplasia, PTH, Endocrinology, Patient, Phase II, Pharmaceutical industry

BUCHAREST, Romania, Jan. 09, 2024 (GLOBE NEWSWIRE) -- Er-Kim , an international pharmaceutical company specializing in the commercialization of novel therapies, today announced it has entered into an exclusive distribution agreement with Danish Company Ascendis Pharma A/S (Nasdaq: ASND) for its endocrinology portfolio.

Key Points: 
  • BUCHAREST, Romania, Jan. 09, 2024 (GLOBE NEWSWIRE) -- Er-Kim , an international pharmaceutical company specializing in the commercialization of novel therapies, today announced it has entered into an exclusive distribution agreement with Danish Company Ascendis Pharma A/S (Nasdaq: ASND) for its endocrinology portfolio.
  • Under the agreement, Er-Kim is appointed as the exclusive Ascendis A/S endocrinology distributor in Albania, Bosnia and Herzegovina, Bulgaria, Croatia, Cyprus, Czechia, Estonia, Greece, Hungary, Kosovo, Latvia, Lithuania, Malta, Montenegro, North Macedonia, Poland, Romania, Serbia, Slovakia, Slovenia, and Türkiye.
  • He added, “Leveraging our exceptional expertise in rare diseases and our preeminent status in endocrinology throughout the region, we eagerly anticipate collaborating with Ascendis and the healthcare community.
  • “We are therefore very pleased to partner with Er-Kim, a company that shares this commitment, to broaden and accelerate access to our innovative Endocrinology Rare Disease treatment options across Central and Eastern Europe and Turkey.”

Specialised Therapeutics signs exclusive agreement with Ascendis Pharma A/S for distribution and commercialisation of three endocrinology therapies in Australia and select South-East Asia countries

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일요일, 1월 7, 2024
Growth hormone, Hypoparathyroidism, Quality of life, Partnership, Achondroplasia, Vitamin D, ACH, European, Disease, Osteochondrodysplasia, Therapy, PTH, Endocrinology, CNP, Patient, Phase II, Pharmaceutical industry

Under the terms of the agreement, ST will commercialise Ascendis Pharma's weekly injectable paediatric human growth hormone treatment SKYTROFATM (lonapegsomatropin), hypoparathyroidism treatment YORVIPATHTM (palopegteriparatide) and investigational achondroplasia therapy TransCon™ CNP (navepegritide).

Key Points: 
  • Under the terms of the agreement, ST will commercialise Ascendis Pharma's weekly injectable paediatric human growth hormone treatment SKYTROFATM (lonapegsomatropin), hypoparathyroidism treatment YORVIPATHTM (palopegteriparatide) and investigational achondroplasia therapy TransCon™ CNP (navepegritide).
  • The agreement spans ST's key regions of Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam.
  • Announcing the partnership, ST Chief Executive Officer Carlo Montagner said this agreement was an important company milestone, signalling ST's expansion into both endocrinology and paediatric medicine.
  • "We are delighted to partner with Specialised Therapeutics to broaden the reach of our endocrinology rare disease portfolio, contributing to our shared goal of making a meaningful difference for patients facing unmet medical needs," she said.

WebMD Announces 16th Annual 2023 Health Heroes

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수요일, 10월 11, 2023
Diagnosis, Neuroscience, COVID-19, Osteochondrodysplasia, Skull, Bias, Family, The Children's Hospital, Machine learning, MS, Headache, COVID, Driving Force, Sickle cell disease, Rickets, Hospital, Bone tumor, Heart Trouble (album), CDC, Genetics, Disease, WHO, Patient, World Health Organization, Chordoma, Movement, Education, Long, CRISPR, HCA Healthcare, Clinical trial, Duke Cancer Institute, Public policy, Duke University, Genetic disorder, King Fahad Specialist Hospital Dammam, Bangladesh Technical Education Board, Jansen, Tissue, Volunteering, Korea Disease Control and Prevention Agency, Grey, Child, National Institutes of Health, Sarah Cannon Research Institute, Life, WebMD, Research, NIH, Cognitive science, Nursing, Pharmaceutical industry, Vaccine, MD, Long COVID

NEWARK, N.J., Oct. 11, 2023 /PRNewswire/ -- WebMD announced today the winners of the 2023 WebMD 16th annual Health Heroes Awards, which includes 4 categories: Lifelong Advocate, Patient Pioneer, Selfless Survivor and Driving Force.

Key Points: 
  • NEWARK, N.J., Oct. 11, 2023 /PRNewswire/ -- WebMD announced today the winners of the 2023 WebMD 16th annual Health Heroes Awards, which includes 4 categories: Lifelong Advocate, Patient Pioneer, Selfless Survivor and Driving Force.
  • "This year's Health Heroes are not only doing amazing things but they also have data or other measurable results that demonstrate the impact of their work," said Dr. John Whyte, Chief Medical Officer of WebMD.
  • With their findings in hand, they began meeting with public health leadership at the Centers for Disease Control and Prevention (CDC), National Institutes of Health (NIH), and World Health Organization (WHO) to forge a path toward more research on Long COVID.
  • For additional information on the 2023 WebMD Health Heroes, click here: https://www.webmd.com/healthheroes/default.htm

BridgeBio Pharma Announces Positive Feedback from the U.S. FDA and EU EMA on the Regulatory Path for a Pivotal Phase 3 Trial of Infigratinib in Children with Achondroplasia

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수요일, 9월 6, 2023
ENDO, Osteochondrodysplasia, FGFR3, Evelina London Children's Hospital, ASHG, Marketing, Pharming, PALO, Aes, Achondroplasia, Șaeș, EMA, Physician, Cancer, European, European Medicines Agency, Growth, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, News, Food, Hypochondroplasia, Safety, Vaccine, Pharmaceutical industry, EU, AHV

”The regulatory feedback clearing the path to approval for infigratinib is great news for children living with achondroplasia and for physicians who treat them.

Key Points: 
  • ”The regulatory feedback clearing the path to approval for infigratinib is great news for children living with achondroplasia and for physicians who treat them.
  • ”We are thrilled to have regulatory alignment on key elements of our Phase 3 trial design and the path towards submitting a marketing application to both the FDA and the EMA.
  • Additionally, BridgeBio expects to initiate clinical development for infigratinib in hypochondroplasia, a skeletal dysplasia closely related to achondroplasia and similarly driven by FGFR3 gain-of-function variants.
  • BridgeBio has previously presented promising preclinical data for hypochondroplasia at ENDO 2023 and ASHG 2022.

Tyra Biosciences Announces FDA Orphan Drug Designation for TYRA-300 for the Treatment of Achondroplasia

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화요일, 8월 1, 2023
Osteochondrodysplasia, FGFR3, FGFR, Achondroplasia, Medical Affairs Bureau, ODD, Quality of life, Mutation, Sleep apnea, Hydrocephalus, Dwarfism, Biology, Diagnosis, Hospital, Rare, FDA, Fibroblast growth factor receptor, Nemours Children's Hospital, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Spinal stenosis, Pharmaceutical industry, Medical device

People living with achondroplasia may experience severe skeletal complications including cranial and spinal stenosis, hydrocephalus and sleep apnea.

Key Points: 
  • People living with achondroplasia may experience severe skeletal complications including cranial and spinal stenosis, hydrocephalus and sleep apnea.
  • TYRA-300 is an oral FGFR3 selective inhibitor whose design may have a meaningful impact on achondroplasia and other skeletal dysplasias.
  • "People living with achondroplasia can have significant health complications that are not adequately addressed with currently available therapies.
  • "The FDA's decision to grant Orphan Drug Designation to TYRA-300 is an important recognition of the potential of our approach to deliver benefit to the achondroplasia community.

BridgeBio Pharma Presents Updated Six Month Results from its Phase 2 Cohort 5 Trial of Infigratinib in Achondroplasia at the Endocrine Society (ENDO) 2023 Annual Conference

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화요일, 6월 20, 2023
ENDO, Osteochondrodysplasia, FGFR3, Partnership, Cohort, PALO, Aes, Infigratinib, Hypoparathyroidism, Saint Andrew's Society, Achondroplasia, Research, IP, Șaeș, Murdoch, Patient, Physician, CXM, Cancer, Endocrine Society, Growth, Conference, Treatment, DSM-IV codes, Hypochondroplasia, Autosome, Pharmaceutical industry, Nursing, AHV

Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source.

Key Points: 
  • Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source.
  • The Phase 2 trial was designed to identify the dose of infigratinib that will be explored in the Phase 3 trial.
  • Our data also presents the opportunity for us to move forward quickly in initiating a Phase 3 clinical trial.
  • Based on the positive results to date, BridgeBio is underway in enrolling children in the run-in for a Phase 3 trial.